PhRMA Report 2012: Medicines in Development for Diabetes

PhRMA Report 2012: Medicines in Development for Diabetes

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  2012 Report Medicines in Development Diabetes presented by america’s biopharmaceutical research companies Biopharmaceutical Research Companies Are Developing More Than 220 Medicines to Treat Diabetes and Related Conditions Each day Medicines in Development more than For Diabetes* 7 million 5,000 American Americans 79 million 130 18.8 million are American adults are Americans diagnosed have been undiagnosed adults with diagnosed have diabetes with prediabetes diabetes America’s biopharmaceutical research com- type 2, in which relative insulin deficiency panies are developing 221 medicines to treat combines with the body failing to properly use diabetes and related conditions. All of the insulin. Between 5 percent and 10 percent 64 medicines in this report are either in clinical of Americans with diabetes have type 1, in trials or awaiting approval by the U.S. Food which the body fails to produce insulin. and Drug Administration. Diabetes affects The medicines in the pipeline today offer nearly 26 million Americans —8.3 percent of hope of reducing the human toll and eco- the U.S. population—and about one-quarter nomic costs of diabetes. Examples of some are unaware they have the disease. medicines now being tested include: 32 Unfortunately, while the death rate due to • medicine that improves A diabetes is declining, the rate of new cases glucose-dependent insulin secretion. has been rising. The number of Americans diagnosed with diabetes has more than tripled • medicine designed to inhibit an enzyme A 14 since 1980, according to the U.S. Centers linked to diabetic neuropathy. for Disease Control and Prevention (CDC). • medicine to treat type 2 diabetes that A Lifestyle choices can affect this increase. may allow for once-weekly dosing. The CDC-led National Diabetes Prevention While diabetes remains a formidable foe, s s ns s ete ete Program found that weight loss and increased ete itio America’s biopharmaceutical research com- iab iab ab physical activity in people at high risk for nd 1D 2D panies are continuing their efforts to develop Di Co diabetes reduced the development of type d novel and more effective therapies to treat the pe pe ifie ted 2 diabetes by 58 percent in a 3-year period. Ty Ty ec ela disease and increase patients’ quality of life. sp Medicines can also help reduce the risk of type s-R Un ete 2 diabetes. For example, one medicine was ab found in studies to lower the risk by 31 percent. Di * Some medicines are listed in more According to the American Diabetes Asso- than one category ciation, most Americans with diabetes have
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status* 11ß-HSD inhibitor Bristol-Myers Squibb diabetes Phase II Princeton, NJ (800) 332-2056 AC 201 Twi Pharmaceuticals type 2 diabetes Phase II (interleukin-1ß inhibitor) Taipei, Taiwan www.twipharma.com AC 165198 Amylin Pharmaceuticals diabetes Phase I (fusion protein) San Diego, CA (858) 552-2200 Biocon Bangalore, India Afrezza® MannKind type 1 diabetes, type 2 diabetes application submitted insulin inhalation Valencia, CA (661) 775-5300 aleglitazar Roche type 2 diabetes Phase III (PPAR a/g agonist) Nutley, NJ (973) 235-5000 -------------------------------------------------- ------------------------------------------- type 2 diabetes Phase I (combination therapy) (973) 235-5000 alogliptin Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted (DPP-IV inhibitor) Deerfield, IL (877) 825-3327 -------------------------------------------------- ------------------------------------------- type 2 diabetes in adolescents and Phase I children (877) 825-3327 alogliptin/metformin Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted (fixed-dose combination) Deerfield, IL (877) 825-3327 alogliptin/pioglitazone Takeda Pharmaceuticals U.S.A. type 2 diabetes application submitted (fixed-dose combination) Deerfield, IL (877) 825-3327 alogliptin/roflumilast Takeda Pharmaceuticals U.S.A type 2 diabetes Phase I Deerfield, IL (877) 825-3327 alpha-1 antitrypsin Omni Bio Pharmaceutical type 1 diabetes Phase I/II Greenwood Village, CO www.omnibiopharma.com AMG 151 Amgen type 2 diabetes Phase II (glucokinase stimulant) Thousand Oaks, CA (800) 772-6436 AMG 876 Amgen type 2 diabetes Phase I (fusion protein) Thousand Oaks, CA (800) 772-6436 *For more information about a specific medicine in this report, please call the telephone number listed. 2 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status anagliptin Kowa Research Institute type 2 diabetes Phase II (DPP-IV antagonist) Morrisville, NC (919) 433-1600 analog insulin-PH20 Halozyme Therapeutics type 1 diabetes, type 2 diabetes Phase II San Diego, CA (858) 794-8889 ARI-2243 Arisaph Pharmaceuticals type 2 diabetes Phase I (DPP-IV antagonist) Boston, MA (617) 986-4500 ASP-1941 Astellas Pharma US type 2 diabetes Phase II (SGLT2 inhibitor) Deerfield, IL (800) 695-4321 AV 133 Avid Radiopharmaceuticals diabetes (diagnosis) Phase I (imaging agent) Philadelphia, PA (215) 298-0700 BGP-15 N-Gene Research Laboratories type 2 diabetes Phase II (JNK inhibitor/insulin sensitiser) New York, NY (212) 605-0225 BI-135585 Boehringer Ingelheim type 2 diabetes Phase I (11ß-HSD1 inhibitor) Pharmaceuticals (800) 243-0127 Ridgefield, CT BIOD-123 Biodel type 1 diabetes Phase II (ultra-rapid-acting recombinant Danbury, CT (203) 796-5000 human insulin) BIOD-125 Biodel type 1 diabetes Phase I completed (ultra-rapid-acting recombinant Danbury, CT (203) 796-5000 human insulin) Byetta® Amylin Pharmaceuticals type 2 diabetes in adolescents Phase III exenatide San Diego, CA (858) 552-2200 CA-18C3 XBiotech type 2 diabetes Phase II Austin, TX (512) 386-2900 canagliflozin Janssen Research Development type 2 diabetes application submitted (SGLT2 inhibitor) Raritan, NJ www.janssenrnd.com canagliflozin/metformin Depomed type 2 diabetes Phase III extended-release Menlo Park, CA (650) 462-5900 (fixed-dose combination) Janssen Research Development www.janssenrnd.com Raritan, NJ canagliflozin/metformin Janssen Research Development type 2 diabetes Phase III immediate-release Raritan, NJ www.janssenrnd.com (fixed-dose combination) Medicines in Development Diabetes 2012 3
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status CAT 1004 Catabasis Pharmaceuticals type 2 diabetes Phase I Cambridge, MA (617) 349-1971 CCX140 ChemoCentryx type 2 diabetes Phase II Mountain View, CA (see also diabetes-related) (650) 210-2900 cetilistat Norgine type 2 diabetes in clinically obese Phase II (lipase inhibitor) Amsterdam, Netherlands patients www.norgine.com CJC-1134-PC ConjuChem type 2 diabetes Phase II (GLP-1 stimulant) Los Angeles, CA www.conjuchem.com CVX 096 Pfizer type 2 diabetes Phase I (PF-04856883) New York, NY (800) 879-3477 (GLP-1 receptor agonist) dapagliflozin AstraZeneca type 2 diabetes application submitted (SGLT2 inhibitor) Wilmington, DE (800) 236-9933 Bristol-Myers Squibb (800) 332-2056 Princeton, NJ ---------------------------------------- ----------------------------------------------- type 2 diabetes Phase III (combination therapy), (800) 236-9933 type 2 diabetes (800) 332-2056 (second-line therapy) dapagliflozin/metformin AstraZeneca type 2 diabetes Phase III (fixed-dose combination) Wilmington, DE (800) 236-9933 Bristol-Myers Squibb (800) 332-2056 Princeton, NJ ------------------------------------------------ --------------------------------- type 2 diabetes Phase I (with extended-release metformin) (800) 236-9933 (800) 332-2056 DB 959 DARA BioSciences type 2 diabetes Phase I (PPAR d/g agonist) Raleigh, NC (919) 872-5578 DC9703 Obio Pharmaceutical type 2 diabetes Phase I (Omega Bio-Pharma) www.obiopharma.com Wilmington, DE diabetes compound Eli Lilly diabetes Phase I Indianapolis, IN (800) 545-5979 diabetes compound Eli Lilly diabetes Phase I Indianapolis, IN (800) 545-5979 diabetes compound Eli Lilly diabetes Phase I Indianapolis, IN (800) 545-5979 4 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status diabetes compound Eli Lilly diabetes Phase I Indianapolis, IN (800) 545-5979 DSP-8658 Sunovion Pharmaceuticals type 2 diabetes Phase I (PPAR a/g agonist) Marlborough, MA (508) 481-6700 dulaglutide Eli Lilly type 2 diabetes Phase III (GLP-1 analog) Indianapolis, IN (800) 545-5979 EGT-0001442 Theracos type 2 diabetes Phase II (SGLT2 inhibitor) Marlborough, MA (508) 688-4221 EGT-0001474 Theracos type 2 diabetes Phase I completed (SGLT2 inhibitor) Marlborough, MA (508) 688-4221 empagliflozin Boehringer Ingelheim type 2 diabetes Phase III (SGLT2 inhibitor) Pharmaceuticals (800) 243-0127 Ridgefield, CT (800) 545-5979 Eli Lilly Indianapolis, IN empagliflozin/linagliptin Boehringer Ingelheim type 2 diabetes Phase III (fixed-dose combination) Pharmaceuticals (800) 243-0127 Ridgefield, CT empagliflozin/metformin Boehringer Ingelheim type 2 diabetes Phase I (fixed-dose combination) Pharmaceuticals (800) 243-0127 Ridgefield, CT emricasan Conatus Pharmaceuticals type 1 diabetes after islet cell Phase I/II San Diego, CA transplantation www.conatuspharma.com encapsulated islet cell ViaCyte type 1 diabetes Phase I/II transplantation therapy San Diego, CA (858) 455-3708 enclomifene Repros Therapeutics type 2 diabetes in men with second- Phase II The Woodlands, TX ary hypogonadism or adult-onset (281) 719-3400 idiopathic hypogonadotropic hypogonadism ertugliflozin/PF-04971729 Pfizer type 2 diabetes Phase II (SGLT2 inhibitor) New York, NY (800) 879-3477 exenatide long-acting Hanmi Pharmaceutical type 2 diabetes Phase II Seoul, South Korea www.hanmipharm.com exenatide suspension Amylin Pharmaceuticals type 2 diabetes Phase II San Diego, CA (858) 552-2200 Medicines in Development Diabetes 2012 5
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status gevokizumab XOMA type 1 diabetes, type 2 diabetes Phase II (IL-1B inhibitor mAb) Berkeley, CA (510) 204-7200 GK1-399 Forest Laboratories type 2 diabetes Phase I/II (TTP-399) New York, NY (800) 947-5227 (glucokinase activator) TransTech Pharma (336) 841-0300 High Point, NC GPR 119 agonist Bristol-Myers Squibb diabetes Phase I Princeton, NJ (800) 332-2056 GSK256073 GlaxoSmithKline type 2 diabetes Phase II (HM74A agonist) Rsch. Triangle Park, NC (888) 825-5249 GSK1070806 GlaxoSmithKline type 2 diabetes Phase I (IL-18 mAb) Rsch. Triangle Park, NC (888) 825-5249 GSK1614235 GlaxoSmithKline type 2 diabetes Phase I (SGLT1 inhibitor) Rsch. Triangle Park, NC (888) 825-5249 GSK2330672 GlaxoSmithKline type 2 diabetes Phase I (ileal bile acid transfer inhibitor) Rsch. Triangle Park, NC (888) 825-5249 HDV-insulin Diasome Pharmaceuticals type 2 diabetes Phase II Conshohocken, PA (609) 923-9443 HE3286 Harbor BioSciences type 2 diabetes Phase II San Diego, CA (858) 587-9333 HIP-2B CureDM type 1 diabetes, type 2 diabetes Phase I (human pro-islet peptide) Wilmington, DE (302) 288-0620 HPP-404 High Point Pharmaceuticals diabetes Phase I (histamine H3 receptor High Point, NC www.highpointpharma.com antagonist) HPP-593 High Point Pharmaceuticals diabetes Phase I (PPAR d agonist) High Point, NC www.highpointpharma.com Humalog® Eli Lilly type 1 diabetes, type 2 diabetes Phase III insulin lispro Indianapolis, IN (800) 545-5979 IBC-VS01 Orban Biotech type 1 diabetes Phase I (insulin B-chain vaccine) Brookline, MA (774) 571-2626 6 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status Ilaris® Novartis type 2 diabetes Phase II/III canakinumab East Hanover, NJ (888) 669-6682 ------------------------------------------------ --------------------------------- type 1 diabetes Phase II (888) 669-6682 indeglitazar Plexxikon type 2 diabetes Phase II (PLX-204) Berkeley, CA (510) 647-4000 INGAP peptide Exsulin type 1 diabetes, type 2 diabetes Phase II Minneapolis, MN www.exsulin.com insulin adjustable basal Biodel diabetes Phase I Danbury, CT (203) 796-5000 insulin glargine Sanofi US type 1 diabetes, type 2 diabetes Phase III (new formulation) Bridgewater, NJ (800) 981-2491 insulin oral sublingual Biodel type 1 diabetes Phase I Danbury, CT (203) 796-5000 insulin transdermal Transdermal Global type 1 diabetes, type 2 diabetes Phase I Broomall, PA www.transdermalglobal.com INT131 InteKrin Therapeutics type 2 diabetes Phase II (PPAR g modulator) Los Altos, CA (650) 941-5501 ISIS-GCCRrx Isis Pharmaceuticals type 2 diabetes Phase I (glucocorticoid receptor Carlsbad, CA (800) 679-4747 antagonist) ISIS-GCGRrx Isis Pharmaceuticals type 2 diabetes Phase I (glucagon receptor antagonist) Carlsbad, CA (800) 679-4747 ISIS-PTP1Brx Isis Pharmaceuticals type 2 diabetes Phase I (PTP-1B gene inhibitor) Carlsbad, CA (800) 679-4747 ISIS-SGLT2rx Isis Pharmaceuticals type 2 diabetes Phase I Carlsbad, CA (800) 679-4747 ITCA 650 Intarcia Therapeutics type 2 diabetes Phase III (exenatide subcutaneous Hayward, CA (510) 782-7800 implant) Janumet® Merck type 2 diabetes in adolescents and Phase III sitagliptin/metformin Whitehouse Station, NJ children (800) 672-6372 Medicines in Development Diabetes 2012 7
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status Januvia® Merck type 2 diabetes in the elderly and Phase III sitagliptin Whitehouse Station, NJ adolescents (800) 672-6372 JNJ-16269110 Janssen Research Development type 2 diabetes Phase II completed (usistapide) Raritan, NJ www.janssenrnd.com JNJ-41443532 Janssen Research Development type 2 diabetes Phase II completed Raritan, NJ www.janssenrnd.com KRP-104 ActivX Biosciences type 2 diabetes Phase II (CD26 antigen inhibitor) La Jolla, CA (858) 558-5558 LCQ908 Novartis type 2 diabetes Phase II (DGAT1 inhibitor) East Hanover, NJ (888) 669-6682 LEZ763 Novartis type 2 diabetes Phase II East Hanover, NJ (888) 669-6682 LIK066 Novartis type 2 diabetes Phase I/II East Hanover, NJ (888) 669-6682 LIM-0705 Limerick Biopharma type 2 diabetes Phase I South San Francisco, CA (650) 742-0110 linagliptin/pioglitazone Boehringer Ingelheim type 2 diabetes Phase III (fixed-dose combination) Pharmaceuticals (800) 243-1027 Ridgefield, CT lisofylline DiaKine Therapeutics type 1 diabetes Phase I (lysophosphatidic acid Norfolk, VA (434) 981-4777 transferase inhibitor) lixisenatide Sanofi US type 2 diabetes Phase III (AVE0010) Bridgewater, NJ (800) 981-2491 lixisenatide/Lantus® insulin Sanofi US type 2 diabetes Phase II glargine Bridgewater, NJ (800) 981-2491 (fixed-dose combination) LX-4211 Lexicon Pharmaceuticals type 2 diabetes Phase II (SLGT1/SLGT2 inhibitor) The Woodlands, TX (281) 863-3000 LY2409021 Eli Lilly type 2 diabetes Phase II (glucagon-R antagonist) Indianapolis, IN (800) 545-5979 8 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status LY2523199 Eli Lilly type 2 diabetes Phase II (11ß-HSD1 inhibitor) Indianapolis, IN (800) 545-5979 LY2605541 Boehringer Ingelheim type 1 diabetes, type 2 diabetes Phase III novel basal insulin/insulin Pharmaceuticals (800) 243-1027 peglispro Ridgefield, CT (800) 545-5979 Eli Lilly Indianapolis, IN LY2963016 Boehringer Ingelheim type 1 diabetes, type 2 diabetes Phase III (new insulin glargine product) Pharmaceuticals (800) 243-1027 Ridgefield, CT (800) 545-5979 Eli Lilly Indianapolis, IN MBX-2982 Metabolex type 2 diabetes Phase II (G-protein coupled receptor) Hayward, CA (510) 293-8800 mesenchymal precursor Mesoblast type 2 diabetes Phase II cell (MPC) product New York, NY (212) 880-2060 (stem cell therapy) metformin delayed-release Elcelyx Therapeutics type 2 diabetes Phase I formulation San Diego, CA (858) 876-1814 (gut sensory modulator) metreleptin Amylin Pharmaceuticals diabetes and/or hypertriglyceridemia application submitted San Diego, CA in patients with inherited or acquired (858) 552-2200 lipodystrophy -------------------------------------------------- ------------------------------------------- type 1 diabetes Phase I (858) 552-2200 mitiglinide Kissei America type 2 diabetes Phase III completed Parsippany, NJ (973) 334-4400 Mitoglitazone™ Metabolic Solutions Development type 2 diabetes Phase II MSDC-0160 Kalamazoo, MI (269) 343-6732 (insulin sensitizer) MK-3102 Merck type 2 diabetes Phase III (DPP-IV inhibitor) Whitehouse Station, NJ (800) 672-6372 MK-5823 Merck type 2 diabetes Phase I Whitehouse Station, NJ (800) 672-6372 MSDC-0602 Metabolic Solutions Development type 2 diabetes Phase II (insulin sensitizer) Kalamazoo, MI (269) 343-6732 Medicines in Development Diabetes 2012 9
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status NN1218 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (insulin aspart fast-acting) Princeton, NJ (800) 727-6500 NN1953 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (insulin oral) Princeton, NJ (800) 727-6500 NN1954 Novo Nordisk type 1 diabetes, type 2 diabetes Phase I (insulin oral) Princeton, NJ (800) 727-6500 NN9068 Novo Nordisk type 2 diabetes Phase III (insulin degludec/liraglutide) Princeton, NJ (800) 727-6500 NN9924 Novo Nordisk type 2 diabetes Phase I (GLP-1 receptor agonist) Princeton, NJ (800) 727-6500 NN9926 Novo Nordisk type 2 diabetes Phase I (GLP-1 stimulant) Princeton, NJ (800) 727-6500 NP-500 Napo Pharmaceuticals type 2 diabetes Phase I San Francisco, CA (see also diabetes-related) (415) 371-8300 OAP-189 Pfizer type 2 diabetes Phase I (PF-05212389) New York, NY (800) 879-3477 Onglyza™ AstraZeneca type 2 diabetes in adolescents and Phase III saxagliptin Wilmington, DE children (800) 236-9933 Bristol-Myers Squibb (800) 332-2056 Princeton, NJ Oral-lyn™ Generex Biotechnology type 1 diabetes Phase III insulin oral Toronto, Canada (416) 364-2551 oxyntomodulin Merck type 2 diabetes Phase I completed Whitehouse Station, NJ (800) 672-6372 PAZ-320 Boston Therapeutics type 2 diabetes Phase II Manchester, NH (978) 886-0421 PB-1023 PhaseBio Pharmaceuticals type 2 diabetes Phase II (GLP-1 receptor agonist) Malvern, PA (610) 891-6500 PEG-FGF-21 Bristol-Myers Squibb diabetes in clinical trials Princeton, NJ (800) 332-2056 10 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status peptide p277 Andromeda Biotech type 1 diabetes (newly diagnosed) Phase III (Orphan Drug) Ness Ziona, Israel www.andromedabio.com PF-04937319 Pfizer type 2 diabetes Phase II New York, NY (800) 879-3477 PF-05175157 Pfizer type 2 diabetes Phase I New York, NY (800) 879-3477 PF-05231023 Pfizer type 2 diabetes Phase I New York, NY (800) 879-3477 Prochymal® Osiris Therapeutics type 1 diabetes Phase II remestemcel-L Columbia, MD (443) 545-1800 Qnexa® VIVUS type 2 diabetes in obese patients Phase II phentermine/topiramate Mountain View, CA (650) 934-5200 Ranexa® Gilead Sciences type 2 diabetes Phase III ranolazine extended-release Foster City, CA (800) 445-3235 RG4929 Roche type 2 diabetes Phase II (11ß-HSD inhibitor) Nutley, NJ (973) 235-5000 RG7201 Roche type 2 diabetes Phase III (SGLT2 inhibitor) Nutley, NJ (973) 235-5000 RG7426 Bayhill Therapeutics type 1 diabetes Phase I completed (BHT-3021) Palo Alto, CA (650) 320-2800 Genentech (800) 626-3553 South San Francisco, CA RG7685 Roche type 2 diabetes Phase II (GLP-1/GIP dual agonist) Nutley, NJ (973) 235-5000 RM-493 Rhythm Pharmaceuticals diabetes Phase I (MC4R peptide) Boston, MA (857) 264-4280 Ryzodeg® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted insulin degludec plus Princeton, NJ (800) 727-6500 insulin aspart S-707106 Shionogi type 2 diabetes Phase II Florham Park, NJ (973) 966-6900 Medicines in Development Diabetes 2012 11
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status semaglutide Novo Nordisk type 2 diabetes Phase II (NN9535) Princeton, NJ (800) 727-6500 sitagliptin/atovastatin Merck type 2 diabetes Phase III (fixed-dose combination) Whitehouse Station, NJ (800) 672-6372 SLX-4090 Kadmon Pharmaceuticals diabetes Phase II (MTP inhibitor) Warrendale, PA (724) 778-6100 SRT2104 Sirtris (GlaxoSmithKline) type 2 diabetes Phase II (SIRT1 protein stimulant) Cambridge, MA (617) 252-6920 SRT2379 Sirtris (GlaxoSmithKline) type 2 diabetes Phase I (SIRT1 protein stimulant) Cambridge, MA (617) 252-6920 SRT3025 Sirtris (GlaxoSmithKline) type 2 diabetes Phase I (SIRT1 protein stimulant) Cambridge, MA (617) 252-6920 Syncria® GlaxoSmithKline type 2 diabetes Phase III albiglutide Rsch. Triangle Park, NC (888) 825-8249 (GLP-1 agonist) T2 18C3 XBiotech type 2 diabetes Phase I (mAb) Austin, TX (512) 386-2900 TAK-329 Takeda Pharmaceuticals U.S.A. type 1 diabetes Phase I (glucokinase stimulant) Deerfield, IL (877) 825-3327 TAK-472 Takeda Pharmaceuticals U.S.A. type 2 diabetes Phase II (DPP-IV antagonist) Deerfield, IL (877) 825-3327 TAK-875 Takeda Pharmaceuticals U.S.A. type 2 diabetes Phase III (GPR40 agonist) Deerfield, IL (877) 825-3327 taurocholic acid Satiogen Pharmaceuticals type 2 diabetes Phase I/II San Diego, CA www.satiogen.com teneligliptin Mitsubishi Pharma America type 2 diabetes Phase I (DPP-IV antagonist) Warren, NJ www.mt-pharma-america.com teplizumab MacroGenics type 1 diabetes Phase III (Orphan Drug) Rockville, MD (301) 251-5172 12 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes, Type 1 and Type 2 Product Name Sponsor Indication Development Status Tresiba® Novo Nordisk type 1 diabetes, type 2 diabetes application submitted insulin degludec Princeton, NJ (800) 727-6500 ------------------------------------------------ --------------------------------- type 1 diabetes in adolescents and Phase III children (800) 727-6500 TT-401 Transition Therapeutics type 2 diabetes Phase I (GLP-1 receptor agonist) Toronto, Canada (416) 260-7770 TTP054 TransTech Pharma type 2 diabetes Phase II (GLP-1 receptor agonist) High Point, NC (336) 841-0300 TTP355 Forest Laboratories type 2 diabetes Phase I (glucokinase stimulant) New York, NY (800) 947-5227 TransTech Pharma (336) 841-0300 High Point, NC TTP814 TransTech Pharma type 2 diabetes Phase I/II (PTP-1ß inhibitor) High Point, NC (336) 841-0300 Victoza® Novo Nordisk type 2 diabetes in adolescents and Phase III liraglutide Princeton, NJ children (800) 727-6500 ------------------------------------------------ --------------------------------- type 1 diabetes Phase II (800) 727-6500 vildagliptin Novartis type 2 diabetes Phase III East Hanover, NJ (888) 669-6682 ------------------------------------------------ --------------------------------- type 2 diabetes in treatment-naïve Phase II/III patients (888) 669-6682 vildagliptin/metformin Novartis type 2 diabetes Phase III completed (fixed-dose combination) East Hanover, NJ (888) 669-6682 VRS-859 Diartis Pharmaceuticals type 2 diabetes Phase I (GLP-1 agonist) Redwood City, CA (650) 963-8595 VVP808 Verva Pharmaceuticals type 2 diabetes Phase II (insulin sensitizer) Carlsbad, CA (760) 271-4783 ZP2929 Boehringer Ingelheim type 2 diabetes Phase I (GLP-1 receptor agonist) Pharmaceuticals (800) 243-0127 Ridgefield, CT www.zealandpharma.com Zealand Pharma Copenhagen, Denmark Medicines in Development Diabetes 2012 13
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  Medicines in Developmentfor Diabetes Diabetes-Related Conditions Product Name Sponsor Indication Development Status ABT-335 Abbott Laboratories diabetic macular edema Phase II completed (choline fenofibrate) Abbott Park, IL (847) 937-6100 ABT-639 Abbott Laboratories diabetic neuropathy Phase II (T-type calcium channel Abbott Park, IL (847) 937-6100 antagonist) ABT-652 Abbott Laboratories diabetic neuropathy Phase II (histamine H3 receptor Abbott Park, IL (847) 937-6100 modulator) ACT-1 topical gel FirstString Research diabetic foot ulcers Phase II Mount Pleasant, SC (843) 860-8372 AKB-9778 Aerpio Therapeutics diabetic macular edema Phase I (HPTPß inhibitor/Tie-2 receptor Cincinnati, OH (513) 985-1920 activator) AL-39,324 Alcon diabetic retinopathy Phase I/II (tyrosine kinase inhibitor) Fort Worth, TX (800) 862-5266 ALG-1001 Allegro Ophthalmics diabetic macular edema Phase I/II (small peptide therapy) San Juan Capistrano, CA (949) 940-8130 AmiKet™ EpiCept diabetic neuropathy Phase II amitriptyline/ketamine Tarrytown, NY (914) 606-3500 ARA 290 Araim Pharmaceuticals diabetic neuropathy Phase II (erythropoietin receptor agonist) Ossining, NY (914) 762-7586 ARC-4558 Aricon Therapeutics diabetic neuropathy Phase II completed (clonidine topical gel) Baltimore, MD (Fast Track) (410) 522-8701 atrasentan Abbott Laboratories diabetic nephropathy Phase II (selective endothelin A receptor Abbott Park, IL (847) 937-6100 antagonist) baricitinib Eli Lilly diabetic nephropathy Phase II (JAK1/JAK2 inhibitor) Indianapolis, IN (800) 545-5979 Incyte Wilmington, DE BVS857 Novartis Pharmaceuticals insulin resistance Phase I East Hanover, NJ (888) 669-6682 C-peptide long-acting Cebix diabetic neuropathy Phase I/II La Jolla, CA (Fast Track) (858) 729-6500 14 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes-Related Conditions Product Name Sponsor Indication Development Status carisbamate Janssen Research Development diabetic neuropathy Phase II completed Raritan, NJ www.janssenrnd.com CCX140 ChemoCentryx diabetic nephropathy Phase II Mountain View, CA (see also diabetes) (650) 210-2900 Cogenzia™ Innocoll diabetic foot ulcers Phase III gentamicin-collagen sponge Ashburn, VA www.innocollinc.com CORT001 Cortendo metabolic syndrome Phase II Halmstad, Sweden www.cortendo.com CTP-499 Concert Pharmaceuticals diabetic nephropathy Phase II Lexington, MA (781) 860-0045 darapladib GlaxoSmithKline diabetic macular edema Phase II Rsch. Triangle Park, NC (888) 825-5249 dextromethorphan/quinidine Avanir Pharmaceuticals diabetic neuropathy Phase III (fixed-dose combination) Aliso Viejo, CA www.avanir.com DSC-127 Derma Sciences diabetic foot ulcers Phase II Princeton, NJ (609) 514-4744 EVK-001 Evoke Pharma diabetic gastroparesis Phase II (intranasal dopamine-receptor San Diego, CA (858) 967-5454 antagonist) Eylea® Bayer HealthCare Pharmaceuticals diabetic macular edema Phase III afilibercept Wayne, NJ (888) 842-2937 Regeneron Pharmaceuticals (914) 847-7000 Tarrytown, NY GRT 6005/06 Forest Laboratories diabetic neuropathy Phase II New York, NY (800) 947-5227 GSK962040 GlaxoSmithKline diabetic gastroparesis Phase II (motilin receptor agonist) Rsch. Triangle Park, NC (888) 825-5249 HO-0303 HealOr diabetic foot ulcers Phase II/III (protein kinase C inhibitor/ Ness Ziona, Israel www.healor.com stimulant) ICO-007 iCo Therapeutics diabetic macular edema Phase II (antisense oligonucleotide) Vancouver, Canada (604) 602-9414 Medicines in Development Diabetes 2012 15
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  Medicines in Developmentfor Diabetes Diabetes-Related Conditions Product Name Sponsor Indication Development Status Iluvien® Alimera Sciences diabetic macular edema application submitted fluocinolone acetonide Alpharetta, GA (Fast Track) (678) 990-5740 pSivida (617) 926-5000 Watertown, MA iroxanadine Orphazyme diabetic foot ulcers, Phase I Copenhagen, Denmark diabetic neuropathy www.orphazyme.com losartan/thioctic acid InVasc Therapeutics diabetic nephropathy Phase II (INV-144) Tucker, GA (678) 736-5900 Lucentis® Genentech diabetic macular edema Phase I ranibizumab implant South San Francisco, CA (800) 626-3553 (sustained delivery) ForSight Vision4 Menlo Park, CA LY2382770 Eli Lilly diabetic nephropathy Phase II (TGF-b mAb) Indianapolis, IN (800) 545-5979 LY2623091 Eli Lilly renal disease associated with Phase II (MR antagonist) Indianapolis, IN diabetes (800) 545-5979 Macugen® Eyetech diabetic macular edema (Fast Track), Phase III pegaptanib Palm Beach Gardens, FL diabetic retinopathy www.eyetech.com MK-6096 Merck diabetic neuropathy Phase II (dual orexin receptor Whitehouse Station, NJ (800) 672-6372 antagonist) modified hepatocyte growth ViroMed diabetic nephropathy Phase I/II factor gene therapy Seoul, South Korea www.viromed.com NanoDOX® Nanotherapeutics diabetic foot ulcers Phase II doxycycline topical gel Alachua, FL (386) 462-9663 nemonoxacin TaiGen Biotechnology infections associated with diabetic Phase II completed Taipei, Taiwan foot ulcers www.taigenbiotech.com Nexagon® CoDa Therapeutics diabetic foot ulcers Phase II antisense oligonucleotide San Diego, CA (858) 677-4074 Nova63035 Novagali Pharma diabetic macular edema Phase I/II (dexamethasone prodrug Evry, France www.novagali.com emulsion) NP-500 Napo Pharmaceuticals metabolic syndrome Phase I San Francisco, CA (see also diabetes) (415) 371-8300 16 Medicines in Development Diabetes 2012
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  Medicines in Developmentfor Diabetes Diabetes-Related Conditions Product Name Sponsor Indication Development Status ocriplasmin ThromboGenics diabetic retinopathy Phase II Iselin, NJ (732) 590-2900 Optina™ Ampio Pharmaceuticals diabetic macular edema Phase II danazol low-dose Greenwood Village, CO (720) 437-6500 Ozurdex® Allergan diabetic macular edema Phase III dexamethasone intravitreal Irvine, CA (714) 246-4500 implant pexiganan Dipexium Pharmaceuticals diabetic foot ulcers application submitted New York, NY (212) 618-1415 PF-655 Quark Pharmaceuticals diabetic macular edema Phase II (RTP801i14) Fremont, CA (510) 402-4020 PF-00489791 Pfizer diabetic nephropathy Phase II (phosphodiesterase 5 inhibitor) New York, NY (800) 879-3477 PF-03882845 Pfizer diabetic nephropathy Phase I New York, NY (800) 879-3477 Pyridorin™ Nephrogenex diabetic nephropathy Phase II pyridoxamine Rsch. Triangle Park, NC (Fast Track) (609) 986-1780 Qutenza® Astellas Pharma US diabetic neuropathy Phase III capsaicin dermal patch Deerfield, IL (800) 695-4321 NeurogesX (650) 358-3300 San Mateo, CA ranirestat Eisai diabetic neuropathy Phase III (AS-3201) Woodcliff Lake, NJ (888) 274-2378 RM-131 Rhythm Pharmaceuticals diabetic gastroparesis Phase II (ghrelin peptide agonist) Boston, MA (857) 264-4280 safotibant (FOV2304) Sanofi US diabetic macular edema Phase II (bradykinin ß1 antagonist) Bridgewater, NJ (800) 981-2491 SAR164653 Sanofi US cardiovascular-related complications Phase I (cathepsin A inhibitor) Bridgewater, NJ and death in diabetic patients (800) 981-2491 SAR407899 Sanofi US diabetic nephropathy Phase I (Rho kinase inhibitor) Bridgewater, NJ (800) 981-2491 Medicines in Development Diabetes 2012 17
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  Medicines in Developmentfor Diabetes Diabetes-Related Conditions Product Name Sponsor Indication Development Status sirolimus ophthalmic MacuSight diabetic macular edema Phase II Union City, CA (subconjunctival) (Fast Track) (510) 400-1440 ------------------------------------------------ --------------------------------- diabetic macular edema Phase I (intravitreous) (510) 400-1440 SKL-NP SK Life Science diabetic neuropathic pain Phase II Fair Lawn, NJ (201) 421-3864 TAK-428 Takeda Pharmaceuticals U.S.A. diabetic neuropathy Phase II (nerve growth factor agonist) Deerfield, IL (877) 825-3327 TTPABC TransTech Pharma diabetic nephropathy Phase I High Point, NC (336) 841-0300 TV1001 TheraVasc diabetic angiopathy Phase II (low-dose sodium nitrate) Cleveland, OH (318) 349-3851 TZP-102 Tranzyme Pharma diabetic gastroparesis Phase II/III (ghrelin agonist) Durham, NC (Fast Track) (919) 474-0020 Vasaloc™ Ampio Pharmaceuticals diabetic nephropathy Phase I danazol low-dose Greenwood Village, CO (720) 437-6500 white blood cell therapy Macrocure diabetic foot ulcers Phase III (intralesional) Tikva, Israel www.macrocure.com M edicines in Development for Diabetes and Diabetes-Related Conditions by Phase of Development* Phase I 85 Phase II 94 Phase III 42 Application Submitted 11 * Some medicines are listed in more than one phase of development. 18 Medicines in Development Diabetes 2012
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  Glossary application submitted—An applicationfor and biological tissues (called ischemia) and It is also one of the most significant long- marketing has been submitted by the company leads to cellular death (necrosis and gangrene, term complications in terms of morbidity and to the Food and Drug Administration (FDA). which in turn may require amputation). Thus, mortality for individual patients with diabetes, tissues which are very sensitive to oxygen which is responsible for up to 40 percent of all diabetes—A chronic disease in which the levels, such as the retina, develop microan- end-stage renal disease (ESRD) cases in the body does not produce or properly use insulin, giopathy and may cause blindness (so-called United States. a hormone that is needed to convert sugar, proliferative diabetic retinopathy). Damage to starches and other food into energy needed nerve cells may cause peripheral neuropathy, diabetic neuropathy—Nerve damage in for daily life. Symptoms may include excessive and to kidney cells, diabetic nephropathy. the arms, hands, legs, and feet caused by thirst, hunger, urination and weight loss. The diabetes. The condition develops slowly and cause of diabetes continues to be a mystery, diabetic gastroparesis—A disorder affecting worsens over time. Depending on the types although both genetics and environmental fac- people with both type 1 and type 2 diabetes of nerves involved, one or more signs and tors such as obesity and lack of exercise ap- in which the stomach takes too long to empty symptoms may be present in diabetic periph- pear to play roles. Type 1 diabetes, the more its contents (delayed gastric emptying). The eral neuropathy. Sensory neuropathy results severe form, results from the body’s failure to vagus nerve controls the movement of food in numbness or tingling in the feet or pain or produce insulin, which “unlocks” the cells of the through the digestive tract. If the vagus nerve discomfort in the feet or legs, including prickly, body, allowing glucose to enter and fuel them. is damaged or stops working, the muscles of sharp pain or burning feet. Motor neuropathy It is estimated that 5 percent to 10 percent of the stomach and intestines do not work nor- involves muscle weakness and loss of muscle Americans who are diagnosed with diabetes mally, and the movement of food is slowed or tone in the feet and lower legs, loss of balance, have type 1, which requires insulin treatment. stopped. Just as with other types of neuropathy or changes in foot shape that can lead to areas Type 2 diabetes results from insulin resistance (nerve damage), diabetes can damage the of increased pressure. Autonomic neuropathy (a condition in which the body fails to properly vagus nerve if blood glucose levels remain results in dry feet and cracked skin. The loss use insulin), combined with relative insulin high over a long period of time. High blood glu- of sensation and other problems associated deficiency. Most Americans who are diagnosed cose causes chemical changes in nerves and with nerve damage make a patient prone to with diabetes have type 2, which in most cases damages the blood vessels that carry oxygen developing skin ulcers (open sores) that can can be controlled if treated properly by a com- and nutrients to the nerves. Gastroparesis can become infected and may not heal. This seri- bination of dietary measures, weight loss, and make diabetes worse by making it more dif- ous complication of diabetes can lead to the oral medication. ficult to manage blood glucose. When food that loss of a foot, a leg, or even a life. has been delayed in the stomach finally enters diabetic angiopathy—Similar to most com- the small intestine and is absorbed, blood diabetic retinopathy—The most common plications brought about by diabetes, diabetic glucose levels rise. If food stays too long in the diabetic eye disease and a leading cause of angiopathy is primarily due to hyperglycemia stomach, it can cause problems like bacterial blindness in American adults. It is caused by or the high levels of blood sugar known as overgrowth because the food has fermented. changes in the blood vessels of the retina, the glucose. Angiopathy can occur in any part Also, the food can harden into solid masses, light sensitive tissue at the back of the eye that of the body where the effect of high glucose called bezoars, that may cause nausea, vomit- is necessary for good vision. In some people levels result in the build up of plaque in the ing, and obstruction in the stomach. Bezoars with diabetic retinopathy, blood vessels may inner walls of the vessels. There are two types can be dangerous if they block the passage of swell and leak fluid. In others, abnormal new of the disease—macroangiopathy or microan- food into the small intestine. blood vessels grow on the surface of the retina. giopathy. In macroangiopathy, atherosclerosis Over time, diabetic retinopathy, which usually and a resultant blood clot forms on the large diabetic nephropathy—Kidney disease or affects both eyes, can worsen and cause blood vessels, sticks to the vessel walls, and damage that can occur in people with diabetes. vision loss. The condition has four stages: 1) blocks the flow of blood. Macroangiopathy may The kidneys have many tiny blood vessels that mild nonproliferative retinopathy, the earliest cause other complications, such as ischemic filter waste from the blood. High blood sugar stage during which microaneurysms occur heart disease, stroke, and peripheral vascular from diabetes can destroy those blood vessels. in the retina’s tiny blood vessels; 2) moder- disease, which contributes to the diabetic foot Over time, the kidney isn’t able to do its job as ate nonproliferative retinopathy, during which ulcers and the risk of amputation. In microan- well and may stop working completely, which some blood vessels that nourish the retina are giopathy, the walls of the smaller blood vessels is called kidney failure. Out of 100 people with blocked; 3) severe nonproliferative retinopathy, become so thick and weak that they bleed, leak diabetes, as many as 40 will develop kidney when many more blood vessels are blocked protein, and slow the flow of blood through the damage. Currently, diabetic nephropathy is and deprive several areas of the retina with body. The decrease of blood flow through clot the leading cause of chronic kidney disease in their blood supply; and 4) proliferative retinopa- formation impairs the flow of oxygen to cells the United States and other Western societies. thy, the advanced stage when signals sent by Medicines in Development Diabetes 2012 19
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  Glossary the retinafor nourishment trigger the growth predisposed to insulin resistance. Acquired Phase I—Researchers test the drug in a small of new blood vessels, which grow along the factors, such as excess body fat and physical group of people, usually between 20 and 80 retina and the surface of the clear, vitreous inactivity, also can elicit insulin resistance. Most healthy adult volunteers, to evaluate its initial gel that fills the inside of the eye. If those thin, people with insulin resistance have abdomi- safety and tolerability profile, determine a fragile blood vessels leak blood, severe vision nal obesity. The biologic mechanisms at the safe dosage range, and identify potential side loss and even blindness can result. Up to 45 molecular level between insulin resistance and effects. percent of Americans diagnosed with diabetes metabolic risk factors aren’t fully understood have some stage of diabetic retinopathy. and appear to be complex. Phase II—The drug is given to volunteer patients, usually between 100 and 300, to see diabetic ulcers—The most common foot macular edema—A condition in which fluid if it is effective, identify an optimal dose, and to injuries leading to lower extremity amputation. can leak into the center of the macula, the part further evaluate its short-term safety. The vast majority of diabetic foot complications of the eye where sharp, straight-ahead vision resulting in amputation begin with the forma- occurs. The fluid makes the macula swell, thus Phase III—The drug is given to a larger, more tion of skin ulcers. The three main causes blurring vision. It can occur at any stage of diverse patient population, often involving be- of diabetic foot ulcers are: neuropathy, poor diabetic retinopathy, although it is more likely tween 1,000 and 3,000 patients (but sometime blood supply, and infection. Up to 15 percent to occur as the disease progresses. About half many more thousands), to generate statistically of diabetics are likely to develop a foot ulcer of the people with proliferative retinopathy also significant evidence to confirm its safety and at some stage in their lives. Early detection have macular edema. effectiveness. They are the longest studies, and appropriate treatment of those ulcers may and usually take place in multiple sites around prevent up to 85 percent of amputations. The metabolic syndrome—A group of meta- the world. risk of lower extremity amputation is 15 to 46 bolic risk factors that raise the risk of type 2 times higher in diabetics than in people who do diabetes in one person, including: abdominal not have the disease. obesity (excessive fat tissue in and around the abdomen); atherogenic dyslipidemia (blood fat insulin resistance—A generalized metabolic disorders—high triglycerides, low HDL choles- disorder caused by the body’s inability to use terol, and high insulin efficiently. Some people are genetically The content of this report has been obtained through industry sources and the Adis “RD Insight” database based on the latest information. Report current as of October 9, 2012. The information may not be comprehensive. For more specific information about a particular product, contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is available on PhRMA’s web site. A publication of PhRMA’s Communications Public Affairs Department. (202) 835-3460 www.phrma.org | www.innovation.org | www.pparx.org | www.buysafedrugs.info Provided as a Public Service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association. Copyright © 2012 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper credit is given. Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Washington, DC 20004 20 Medicines in Development Diabetes 2012
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  Selected Facts AboutDiabetes in the United States* Diabetes1 • In the United States, 25.8 million people, or nearly 8.3 percent of the population, have diabetes. An estimated 18.8 million have been diagnosed, but 7 million people are not aware that they have the disease. Another 79 million have pre-diabetes. • In 2010, 1.9 million new cases of diabetes were diagnosed in people ages 20 and older. In that age group, 11.3 percent, or 25.6 million people, have diabetes—13 million men and 12.6 million women. • In young people under the age of 20, 215,000, or 0.26 percent, have diabetes. About 1 in every 400 children and adolescents has diabetes. Non-Hispanic white youth have the highest rate of new cases of type 1 diabetes (24.8 per 100,000 per year among those younger than age 10 and 22.6 per 100,000 per year among those ages 10–19). • Among those people age 65 years and older, 10.9 million, or 26.9 percent, have diabetes. In 2004, heart disease was noted on 68 percent and stroke on 16 percent of diabetes-related death certificates among that age group. • The 2007-2009 national survey data for people diagnosed with diabetes ages 20 and older found the following prevalence of diabetes by race and ethnicity: 7.1 percent of non-Hispanic whites, 8.4 percent of Asian Americans, 12.6 percent of non-Hispanic blacks, and 11.8 percent of Hispanics. Among Hispanics, the prevalence rates were 7.6 percent for Cubans, 13.3 percent for Mexican Americans, and 13.8 percent for Puerto Ricans. • Diabetes is the seventh leading cause of death in the United States. In 2007, diabetes was listed as the underlying cause on 71,382 death certifi- cates and as a contributing factor on an additional 160,022 death certificates, which means that diabetes contributed to a total of 231,404 deaths. Diabetes-Related Conditions • Each year, about 1 percent to 4 percent of people with diabetes develop a foot ulcer, and 10 percent to 15 percent of those with diabetes will have at least one foot ulcer during their lifetimes. Lower extremity amputations (LEAs) are an extreme complication associated with diabetes and foot ulcers. In the United States, nearly 80,000 LEAs are performed on diabetics each year. In 2005, the overall rate of hospital discharges for new LEAs was about 4.3 per 1,000 people with diabetes compared with a rate of about 0.3 per 1,000 in the general population.2 • Adults with diabetes have heart disease death rates and stroke risk at about 2 to 4 times higher than adults without diabetes. In 2004, heart disease was noted on 68 percent of diabetes-related death certificates among people ages 65 years and older, while stroke was noted on 16 percent of diabetes-related death certificates among that age group.1 • Diabetes is the leading cause of kidney failure, accounting for 44 percent of new cases in 2008. That year, 48,374 people with diabetes began treatment for end-stage kidney disease, and another 202,290 people with end-stage kidney disease due to diabetes were living on chronic dialysis or with a kidney transplant in the United States.1 • About 60 percent to 70 percent of people with diabetes have mild to severe forms of nervous system damage (diabetic neuropathy).1 • Diabetes is the leading cause of new cases of blindness among adults ages 20–74. From 2005-2008, 4.2 million people with diabetes (28.5 percent) age 40 and older had diabetic retinopathy, and of those, almost 0.7 million (4.4 percent) had advanced diabetic retinopathy that could lead to severe vision loss.1 Costs1 • The total annual economic cost of diabetes in 2007 was $174 billion. Direct medical costs totaled $116 billion, while indirect costs (including disability, work loss, and premature mortality) came to $58 billion. If the additional costs of undiagnosed diabetes ($18 billion), prediabetes ($25 billion), and gestational diabetes ($623 million) were factored in, the total cost of diabetes in the United States adds up to $218 billion. • Average medical expenditures among people with diagnosed diabetes are 2.3 times higher than expenditures for people who don’t have diabetes. Sources: 1. American Diabetes Association, www.diabetes.org 2. Agency for Healthcare Research and Quality, www.ahrq.gov Medicines in Development Diabetes 2012 21
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  The Drug Discovery,Development and Approval Process Developing a new medicine takes an average of 10-15 years; For every 5,000-10,000 compounds in the pipeline, only 1 is approved. The Drug Development and Approval Process The U.S. system of new drug approvals is in people. The IND shows results of previous statistically significant evidence to confirm its perhaps the most rigorous in the world. experiments; how, where and by whom the safety and effectiveness. They are the longest new studies will be conducted; the chemical studies, and usually take place in multiple sites It takes 10-15 years, on average, for an structure of the compound; how it is thought around the world. experimental drug to travel from lab to U.S. to work in the body; any toxic effects found in patients, according to the Tufts Center for the New Drug Application (NDA)/Biologic the animal studies; and how the compound Study of Drug Development. Only five in 5,000 License Application (BLA). Following the is manufactured. All clinical trials must be compounds that enter preclinical testing make completion of all three phases of clinical trials, reviewed and approved by the Institutional it to human testing. And only one of those five a company analyzes all of the data and files an Review Board (IRB) where the trials will be is approved for sale. NDA or BLA with FDA if the data successfully conducted. Progress reports on clinical trials demonstrate both safety and effectiveness. On average, it costs a company $1.2 billion, must be submitted at least annually to FDA and The applications contain all of the scientific including the cost of failures, to get one new the IRB. information that the company has gathered. medicine from the laboratory to U.S. patients, Clinical Trials, Phase I—Researchers test Applications typically run 100,000 pages or according to a 2007 study by the Tufts Center the drug in a small group of people, usually more. for the Study of Drug Development. between 20 and 80 healthy adult volunteers, to Approval. Once FDA approves an NDA or Once a new compound has been identified in evaluate its initial safety and tolerability profile, BLA, the new medicine becomes available the laboratory, medicines are usually devel- determine a safe dosage range, and identify for physicians to prescribe. A company must oped as follows: potential side effects. continue to submit periodic reports to FDA, Preclinical Testing. A pharmaceutical com- Clinical Trials, Phase II—The drug is given including any cases of adverse reactions and pany conducts laboratory and animal studies to volunteer patients, usually between 100 and appropriate quality-control records. For some to show biological activity of the compound 300, to see if it iseffective, identify an optimal medicines, FDA requires additional trials against the targeted disease, and the com- dose, and to further evaluate its short-term (Phase IV) to evaluate long-term effects. pound is evaluated for safety. safety. Discovering and developing safe and effective Investigational New Drug Application (IND). Clinical Trials, Phase III—The drug is given to new medicines is a long, difficult, and expensive After completing preclinical testing, a company a larger, more diverse patient population, often process. PhRMA member companies invested files an IND with the U.S. Food and Drug involving between 1,000 and 3,000 patients (but an estimated $49.5 billion in research and Administration (FDA) to begin to test the drug sometime many more thousands), to generate development in 2011.