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PhRMA Report 2012: Medicines in Development for Alzheimers
 

PhRMA Report 2012: Medicines in Development for Alzheimers

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    PhRMA Report 2012: Medicines in Development for Alzheimers PhRMA Report 2012: Medicines in Development for Alzheimers Document Transcript

    • 2012 ReportMedicines in DevelopmentAlzheimer’s Diseasepresented by america’s biopharmaceuticalresearch companies Biopharmaceutical Research Companies are Developing Nearly 100 Medicines for Alzheimer’s Disease and Other DementiasMedicines in DevelopmentFor Alzheimer’s Disease* 81 Today, more than 5 million Americans are suf- human clinical trials or awaiting U.S. Food and fering from Alzheimer’s disease. The disease Drug Administration (FDA) review. They are ravages the minds of patients, crushes entire exploring various new approaches to treating families and currently costs the health care Alzheimer’s disease, including: system $200 billion a year. These sobering •  medicine that inhibits the formation and A statistics are projected to get much worse as accumulation of amyloid-beta and tau the 76 million American baby boomers age. protein deposits. If no new medicines are found to prevent, •  n intranasal medicine that is able to A delay or stop the progression of Alzheimer’s penetrate the blood-brain barrier for mild disease, the number of people affected in cognitive impairment, a precursor to America will jump to 13.5 million by 2050, Alzheimer’s. according to the Alzheimer’s Association. Costs for care for Alzheimer’s patients will • A gene therapy for the treatment of  increase five-fold to $1.08 trillion a year. Alzheimer’s disease. Even modest progress can drastically change The quest is intense and financially risky. It this trajectory. A breakthrough medicine that takes, on average, more than $1 billion and 11 delays the onset of Alzheimer’s disease by 10 to 15 years to develop a new medicine. But just five years could decrease the number of new scientific advances are increasing our 5 Americans suffering from the disease in 2050 knowledge, and researchers are using every 2 by 43 percent and the related costs of care by cutting-edge tool at their disposal. With contin- $447 billion. Hope for the future lies in medical ued dedication, we hope to make a difference for every person at risk of suffering from this se ’s cs so on as e innovation. s as Di mer er sti nti Di iti terrible, debilitating disease. rd gn no me ei America’s biopharmaceutical companies cur- Co ag zh De Di Al rently have 93 medicines in development for* Some medicines are in development Alzheimer’s disease and dementias—either infor more than one disorder.
    • Medicines in Development for Alzheimer’s Disease Alzheimer’s Disease and Dementias Product Name Sponsor Indication Development Status* AAB-002 Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase 0 (amyloid beta-protein inhibitor South San Francisco, CA (888) 381-4595 mAb) Pfizer (800) 879-3477 New York, NY AAB-003/PF-05236812 Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase I (amyloid beta-protein inhibitor South San Francisco, CA (888) 381-4595 mAb) Pfizer (800) 879-3477 New York, NY ABT-126 Abbott Laboratories mild to moderate Alzheimer’s disease Phase II (alpha-7 neuronal nicotinic Abbott Park, IL (847) 937-6100 receptor antagonist) -------------------------------------------------- ------------------------------------------- Alzheimer’s disease Phase I (combination therapy), (847) 937-6100 Alzheimer’s disease (elderly) ABT-288 Abbott Laboratories Alzheimer’s disease Phase II (neurotransmitter receptor Abbott Park, IL (847) 937-6100 modulator) ABT-384 Abbott Laboratories mild to moderate Alzheimer’s disease Phase II Abbott Park, IL (847) 937-6100 ABT-560 Abbott Laboratories cognitive disorders Phase I (alpha-4 beta-2 nicotinic Abbott Park, IL (847) 937-6100 receptor modulators) ABT-957 Abbott Laboratories Alzheimer’s disease Phase I (calpain inhibitor) Abbott Park, IL (847) 937-6100 ACC-002 Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase 0 (amyloid-beta peptide South San Francisco, CA (888) 381-4595 conjugate) Pfizer (800) 879-3477 New York, NY AD02 vaccine Affiris Alzheimer’s disease Phase II Vienna, Austria (888) 825-5249 GlaxoSmithKline Rsch. Triangle Park, NC AD03 vaccine Affiris Alzheimer’s disease Phase I Vienna, Austria (888) 825-5249 GlaxoSmithKline Rsch. Triangle Park, NC*For more information about a specific medicine in this report, please call the telephone number listed.2 Medicines in Development Alzheimer’s Disease 2012
    • Medicines in Development for Alzheimer’s Disease Alzheimer’s Disease and Dementias Product Name Sponsor Indication Development Status ADS-8704 Adamas Pharmaceuticals moderate to severe Alzheimer’s Phase II (donepezil/memantine) Emeryville, CA disease (510) 450-3500 APH-0703 Aphios Alzheimer’s disease, Phase I/II Woburn, MA cognitive disorders (781) 932-6933 ARC029 Archer Pharmaceuticals mild to moderate Alzheimer’s disease Phase I (soluble amyloid Sarasota, FL (941) 755-6644 reducing/clearing agent) (Orphan Drug) ARC031 Archer Pharmaceuticals Alzheimer’s disease Phase I (soluble amyloid Sarasota, FL (941) 755-6644 reducing/clearing agent) ASP0777 Astellas Pharma US dementia associated with Alzheimer’s Phase I Deerfield, IL disease (800) 695-4321 AVN 101 Avineuro Pharmaceuticals cognitive enhancer in Alzheimer’s Phase II (serotonin 6 receptor San Diego, CA disease (858) 436-1537 antagonist) AVN 322 Avineuro Pharmaceuticals Alzheimer’s disease Phase I (serotonin 6 receptor San Diego, CA (858) 436-1537 antagonist) AVN 397 Avineuro Pharmaceuticals Alzheimer’s disease Phase II San Diego, CA (858) 436-1537 AZD1446 AstraZeneca Alzheimer’s disease Phase I (alpha4/beta2 neuronal Wilmington, DE (800) 236-9933 nicotinic receptor agonist) Targacept (336) 480-2100 Winston-Salem, NC AZD3480 AstraZeneca Alzheimer’s disease Phase II (ispronicline) Wilmington, DE (800) 236-9933 Targacept Winston-Salem, NC AZD4694 Navidea Biopharmaceuticals Alzheimer’s disease (diagnosis) Phase II (fluorine-18 labeled precision Dublin, OH (614) 793-7500 radiopharmaceutical) AZD5213 AstraZeneca Alzheimer’s disease Phase II (histamine-3 receptor Wilmington, DE (800) 236-9933 antagonist)Medicines in Development Alzheimer’s Disease 2012 3
    • Medicines in Development for Alzheimer’s DiseaseALZHEIMER’S DISEASE AND DEMENTIASProduct Name Sponsor Indication Development Statusß secretase inhibitor Eli Lilly Alzheimer’s disease Phase II Indianapolis, IN (800) 545-5979BAN2401 BioArtic Neuroscience mild to moderate Alzheimer’s disease Phase I(amyloid beta-protein inhibitor) Stockholm, Sweden www.bioarti.se Eisai (888) 274-2378 Woodcliff Lake, NJbapineuzumab subcutaneous Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase II(AAB-001) South San Francisco, CA (subcutaneous) (888) 381-4595 Pfizer (800) 879-3477 New York, NYBCI-632 BrainCells Alzheimer’s disease Phase I San Diego, CA (858) 812-7700BCI-838 BrainCells Alzheimer’s disease Phase I San Diego, CA (858) 812-7700BIIB037 Biogen Idec Alzheimer’s disease Phase I(amyloid beta-protein inhibitor) Cambridge, MA (617) 679-2000bisnorcymserine QR Pharma Alzheimer’s disease Phase I(BNC) Berwyn, PA (610) 727-3913BMS-241027 Bristol-Myers Squibb Alzheimer’s disease, tauopathies Phase I(microtubule stabilizer) Princeton, NJ (800) 332-2056BMS-708163 Bristol-Myers Squibb Alzheimer’s disease Phase II(avagacestat) Princeton, NJ (800) 332-2056BMS-932481 Bristol-Myers Squibb Alzheimer’s disease Phase I(gamma secretase modulator) Princeton, NJ (800) 332-2056BMS-933043 Bristol-Myers Squibb cognitive impairment Phase I(a-7 nicotinic agonist) Princeton, NJ (800) 332-2056CAD106 Novartis Pharmaceuticals Alzheimer’s disease Phase II(amyloid beta-protein inhibitor) East Hanover, NJ (888) 669-6682CERE-110 Ceregene Alzheimer’s disease Phase II(AAV-NGF gene therapy) San Diego, CA (858) 458-8800CHF-5074 Chiesi Pharmaceuticals mild cognitive impairment Phase II(amyloid precursor protein Rockville, MD (301) 424-2661secretase modulator)4 Medicines in Development Alzheimer’s Disease 2012
    • Medicines in Development for Alzheimer’s Disease Alzheimer’s Disease and Dementias Product Name Sponsor Indication Development Status crenezumab Genentech Alzheimer’s disease Phase II (anti-Abeta) South San Francisco, CA (800) 626-3553 CTS-21166 Astellas Pharma US Alzheimer’s disease Phase I (ß-secretase inhibitor) Deerfield, IL (800) 695-4321 CoMentis (650) 359-2600 South San Francisco, CA CX717 Cortex Pharmaceuticals Alzheimer’s disease Phase II completed Irvine, CA (949) 727-3157 davunetide intranasal Allon Therapeutics Alzheimer’s disease, mild cognitive Phase II Vancouver, Canada impairment (604) 736-0634 docosahexaenoic acid Martek Biosciences Alzheimer’s disease Phase III (DHA) Parsippany, NJ (973) 257-8011 DSP-8658 Sunovion Pharmaceuticals Alzheimer’s disease Phase I (PPAR a/g agonist) Marlborough, MA (508) 481-6700 E2212 Eisai Alzheimer’s disease Phase I (amyloid precursor protein Woodcliff Lake, NJ (888) 274-2378 secretase modulator) E2609 Eisai Alzheimer’s disease Phase I (BACE1 protein inhibitor) Woodcliff Lake, NJ (888) 274-2378 ELND005 Elan mild to moderate Alzheimer’s disease Phase II (amyloid beta-protein inhibitor) South San Francisco, CA (Fast Track) (650) 877-0900 Transition Therapeutics (416) 260-7770 Toronto, Canada EVP-0962 EnVivo Pharmaceuticals Alzheimer’s disease Phase I (amyloid precursor protein Watertown, MA (617) 225-4250 secretase modulator) EVP-6124 EnVivo Pharmaceuticals mild to moderate Alzheimer’s disease Phase II (α7-nAChR agonist) Watertown, MA (617) 225-4250 Exebryl-1® ProteoTech Alzheimer’s disease Phase I Kirkland, WA (425) 823-0400 F18-florbetaben Piramal Healthcare Alzheimer’s disease (diagnosis) Phase III (molecular imaging agent) Mumbai, India www.piramalhealthcare.com F18-flutemetamol GE Healthcare Alzheimer’s disease (diagnosis) Phase III (PET imaging agent) Waukesha, WI www.gehealthcare.comMedicines in Development Alzheimer’s Disease 2012 5
    • Medicines in Development for Alzheimer’s DiseaseAlzheimer’s Disease and DementiasProduct Name Sponsor Indication Development StatusGammagard® Baxter Healthcare early-stage Alzheimer’s disease, Phase IIIimmune globulin intravenous Deerfield, IL mid-stage Alzheimer’s disease (800) 422-9837(human), 10% solutiongantenerumab Roche prodromal Alzheimer’s disease Phase II/III(RG1450) Nutley, NJ (973) 235-5000GSK239512 GlaxoSmithKline Alzheimer’s disease Phase II completed Rsch. Triangle Park, NC (888) 825-5249GSK742457 GlaxoSmithKline Alzheimer’s disease Phase II completed(5HT6 antagonist) Rsch. Triangle Park, NC (888) 825-5249GSK933776A GlaxoSmithKline Alzheimer’s disease Phase I completed(anti-B amyloid mAb) Rsch. Triangle Park, NC (888) 825-5249HPP-854 High Point Pharmaceuticals Alzheimer’s disease Phase I(BACE1 inhibitor) High Point, NC (336) 841-0300human immunoglobulin Grifols USA Alzheimer’s disease Phase III(intravenous) Los Angeles, CA (888) 474-3657immune globulin high dose Octapharma USA Alzheimer’s disease (elderly) Phase II completed Hoboken, NJ (201) 604-1130irdabisant Cephalon cognitive dysfunction associated with Phase I(CEP-26401) Frazer, PA Alzheimer’s disease (610) 344-0200LMTX TauRx Pharmaceuticals mild to moderate Alzheimer’s disease Phase I(TRx-0237) Singapore www.taurx.comLNK-754 Link Medicine mild Alzheimer’s disease Phase I completed Waltham, MA (781) 577-6700LU AE58054 Lundbeck Alzheimer’s disease Phase II Deerfield, IL (800) 455-1141MCD-386/glycopyrrolate Mithridion autosomal dominant Alzheimer’s Phase I Madison, WI disease www.mithridion.comMK-3134 Merck dementia Phase I completed Whitehouse Station, NJ (800) 672-6372MK-3328 Merck Alzheimer’s disease (diagnosis) Phase I completed(PET tracer) Whitehouse Station, NJ (800) 672-6372MK-8931 Merck Alzheimer’s disease Phase I(BACE1 inhibitor) Whitehouse Station, NJ (800) 672-63726 Medicines in Development Alzheimer’s Disease 2012
    • Medicines in Development for Alzheimer’s Disease Alzheimer’s Disease and Dementias Product Name Sponsor Indication Development Status MSDC-0160 Metabolic Solutions Development Alzheimer’s disease Phase II Company (269) 343-6732 Kalamazoo, MI NIC5-15 Humanetics Alzheimer’s disease Phase II Minneapolis, MN (952) 937-7660 PF-05212377 Pfizer Alzheimer’s disease Phase I (SAM-760) New York, NY (800) 879-3477 pioglitazone Takeda Pharmaceuticals U.S.A. Alzheimer’s disease (diagnosis) Phase I companion Deerfield, IL (877) 825-3327 diagnostic Zinfadel Pharmaceuticals Chapel Hill, NC Posiphen™ QR Pharma Alzheimer’s disease, Phase II R-phenserine Berwyn, PA mild cognitive impairment (610) 727-3913 PRX-3140 Nanotherapeutics Alzheimer’s disease Phase II (5-HT4 partial agonist) Alachua, FL (386) 462-9663 RG1577 Roche Alzheimer’s disease Phase II (MAO-B inhibitor) Nutley, NJ (973) 235-5000 RG1662 Roche cognitive disorders Phase I (GABAA a5 receptor modulator) Nutley, NJ (973) 235-5000 RG7129 Roche Alzheimer’s disease Phase I (BACE1 protein inhibitor) Nutley, NJ (973) 235-5000 rilapladib GlaxoSmithKline Alzheimer’s disease Phase II Rsch. Triangle Park, NC (888) 825-5249 Human Genome Sciences (301) 309-8504 Rockville, MD RVX-208 Resverlogix Alzheimer’s disease Phase I (BET protein inhibitor) Calgary, Canada (403) 254-9252 SAR110894 Sanofi US Alzheimer’s disease Phase II (H3 antagonist) Bridgewater, NJ (800) 981-2491 SAR228810 Sanofi US Alzheimer’s disease Phase I Bridgewater, NJ (800) 981-2491 sGC-1061 sGC Pharma Alzheimer’s disease Phase I Wellesley, MA (613) 791-4464Medicines in Development Alzheimer’s Disease 2012 7
    • Medicines in Development for Alzheimer’s DiseaseAlzheimer’s Disease and DementiasProduct Name Sponsor Indication Development Statussolanezumab Eli Lilly Alzheimer’s disease Phase III Indianapolis, IN (800) 545-5979ST-101 Sonexa Therapeutics Alzheimer’s disease Phase II San Diego, CA (858) 356-6250SYN-120 Biotie Therapies cognitive disorders associated with Phase I completed South San Francisco, CA Alzheimer’s disease (650) 244-4850T-817MA Toyama Chemical mild to moderate Alzheimer’s disease Phase II completed Tokyo, Japan www.toyama-chemical.co.jpTC-5619 Targacept Alzheimer’s disease Phase I Winston-Salem, NC (336) 480-2100TD-8954 Theravance cognitive impairment associated with Phase I(5-HT4 agonist) South San Francisco, CA Alzheimer’s disease (877) 275-8479TTP-448 TransTech Pharma Alzheimer’s disease Phase II(RAGE antagonist) High Point, NC (336) 841-0300UB-311 United Biomedical mild to moderate Alzheimer’s disease Phase I(amyloid beta protein inhibitor Hauppauge, NY (631) 273-2828vaccine)V950 Merck Alzheimer’s disease Phase Ivaccine Whitehouse Station, NJ (800) 672-6372vanutide cridificar Janssen Alzheimer Immunotherapy Alzheimer’s disease Phase II(ACC-001) South San Francisco, CA (888) 381-4595 Pfizer (800) 879-3477 New York, NYvelusetrag Theravance Alzheimer’s disease Phase I(TD-5108) South San Francisco, CA (877) 275-8479VI-1121 VIVUS Alzheimer’s disease Phase II Mountain View, CA (650) 934-5200XEL 001HP Xel Pharmaceuticals Alzheimer’s disease Phase I(transdermal patch) Draper, UT (866) 832-75468 Medicines in Development Alzheimer’s Disease 2012
    • GlossaryAlzheimer’s disease—The most common review of drugs to treat serious diseases and delusions, and paranoia, and may behaveform of dementia, characterized by progressive fill an unmet medical need. The purpose is to impulsively.and chronic deterioration of cognitive functions, get new drugs for serious diseases to patientsincluding memory, thinking and reasoning. earlier and must be requested by the biophar- PET imaging—Positron emission tomographyEarly manifestations include forgetfulness, maceutical company. Fast Track addresses a (PET), a noninvasive medical imaging tech-impaired ability to focus, and changes in mood broad range of serious diseases. Generally, nique that utilizes a radioactive agent (“tracer”)and personality. As the disease progresses, determining factors include whether the drug incorporated in a biologically active moleculethere is a loss of computational ability, in addi- will have an impact on such factors as survival, and a scanner to produce three-dimensionaltion to word-finding problems and difficulty with day-to-day functioning, or the likelihood that images of the body.ordinary activities. Ultimately, the disease leads the disease, if left untreated, will progress Phase 0—First-in-human trials conductedto severe memory loss, complete disorienta- from a less severe condition to a more serious in accordance with FDA’s 2006 guidance ontion, social withdrawal, loss of independence, one. Filling an unmet medical need is defined exploratory Investigational New Drug (IND)and is fatal. as providing a therapy where none exists or studies designed to speed up development of providing a therapy which may be potentially promising drugs by establishing very early onapplication submitted—An application for superior to existing therapy. Once a drugmarketing approval has been submitted to the whether the agent behaves in human subjects receives Fast Track designation, early and as was anticipated from preclinical studies.U.S. Food and Drug Administration (FDA). frequent communication between the FDA andThe application can either be an NDA (new a drug company is encouraged throughout the Phase I—Researchers test the drug in a smalldrug application) or a BLA (biologic license entire drug development and review process. group of people, usually between 20 and 80application). The frequency of communication assures that healthy adult volunteers, to evaluate its initial questions and issues are resolved quickly, with safety and tolerability profile, determine acognitive disorders—Disorders of the higher the goal to achieve earlier drug approval and safe dosage range, and identify potential sidemental processes, including understand- access by patients. effects.ing, reasoning, knowledge, and intellectualcapacity. A person with a cognitive disorder, mild Alzheimer’s disease—A stage of Al- Phase II—The drug is given to volunteersuch as Alzheimer’s disease, does not process zheimer’s disease characterized by a series of patients, usually between 100 and 300, to seeinformation correctly within the brain, resulting changes in cognitive abilities that may include, if it is effective, identify an optimal dose, and toin impaired awareness and judgment, difficulty memory loss for recent events, difficulty with further evaluate its short-term safety.reasoning and focusing, loss of memory and problem solving, changes in personality, dif-abnormal mental capacity. People with cogni- ficulty organizing and expressing thoughts, Phase III—The drug is given to a larger, moretive disorders have problems acquiring, men- getting lost or misplacing belongings. This is diverse patient population, often involving be-tally organizing and responding to information, the stage at which the disease is often first tween 1,000 and 3,000 patients (but sometimewhich results in an inability to function normally diagnosed. many more thousands), to generate statisticallyin everyday life situations. significant evidence to confirm its safety and moderate Alzheimer’s disease—A stage effectiveness. They are the longest studies,dementia—Loss of mental ability that inter- of Alzheimer’s disease characterized by and usually take place in multiple sites aroundferes with normal daily activities. It lasts more increased confusion, greater memory loss, sig- the world.than six months, it not present at birth and is nificant changes in personality, and the neednot associated with loss or altered conscious- for assistance with basic daily activities. These tauopathies—A group of neurodegenerativeness. The natural decline of these functions changes are related to damage in areas of the diseases characterized by accumulation ofwith age is grossly exaggerated in dementia. brain that control language, reasoning, sensory tau (τ) protein in the brain. Those diseases processing, and conscious thinking. At this include Alzheimer’s disease, Pick’s disease,Fast Track—A U.S. Food and Drug Admin- corticobasal degeneration, and other related stage, patients may have problems recognizingistration (FDA) process designed to facilitate disorders. family and friends, experience hallucinations,development and expedite the regulatoryMedicines in Development Alzheimer’s Disease 2012 9
    • Selected Facts about Alzheimer’s Disease and Other Dementias Alzheimer’s Disease/Dementias • An estimated 5.4 million Americans have Alzheimer’s disease (AD)—the most common form of dementia—including some 200,000 people younger than age 65 who make up the younger-onset AD population. • Some 5.2 million people with Alzheimer’s are age 65 and older; of these, 3.4 million are women and 1.8 million are men. •  accounts for 60 percent to 80 percent of all cases of dementias. AD • In 2010, an estimated 5.1 million Americans age 65 and older had Alzheimer’s disease. That number was expected to increase to 5.6 million in 2020; 7.8 million by 2030; and 13.5 million by 2050. By that year, the percentage of people age 65 and older with Alzheimer’s could be as high as 16 percent unless researchers find a way to prevent or treat the disease. •  is the sixth leading cause of death in the United States and the fifth leading cause of death in Americans age 65 and older. The proportion AD of deaths due to AD has risen significantly. Between 2000 and 2008, the proportion of deaths due to heart disease, stroke, and prostate cancer decreased by 13 percent, 20 percent, and 8 percent, respectively, whereas the proportion due to AD increased by 66 percent. • In 2009, Alzheimer’s was listed as the cause of death for 79,003 Americans. In 2000, only 49,044 death certificates recorded Alzheimer’s as the underlying cause. This increase could be due to reporting changes and an increase in actual Alzheimer’s deaths. • Some 60 percent to 70 percent of people with Alzheimer’s and other dementias live at home, where they are cared for by family and friends. • In 2011, more than 15 million family members and other unpaid caregivers provided an estimated 17.4 billion hours of care to people with AD and other dementias, a contribution valued at more than $210 billion. • More than 70 percent of family and other unpaid caregivers of people with Alzheimer’s disease and other dementias are women. • Medicare payments for services to beneficiaries age 65 and older with AD and other dementias are three times as great as payments for beneficiaries without those conditions, and Medicaid payments are 19 times as great. In 2012, payments for health care, long-term care, and hospice services for people age 65 and older with AD and other dementias are expected to be $200 billion (not including the contributions of unpaid caregivers). Source: Alzheimer’s Association, 2012 Alzheimer’s Disease Facts and Figures. The content of this report has been obtained through industry sources and the Adis “R&D Insight” database based on the latest information. Report current as of August 15, 2012. The information may not be comprehensive. For more specific information about a particular product, con- tact 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 2000410 Medicines in Development Alzheimer’s Disease 2012
    • 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 ProcessThe U.S. system of new drug approvals is in people. The IND shows results of previous statistically significant evidence to confirm itsperhaps 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 sitesIt 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 inpatients, according to the Tufts Center for the New Drug Application (NDA)/Biologic the animal studies; and how the compoundStudy of Drug Development. Only five in 5,000 License Application (BLA). Following the is manufactured. All clinical trials must becompounds that enter preclinical testing make completion of all three phases of clinical trials, reviewed and approved by the Institutionalit 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 beis 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 scientificincluding 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 oraccording 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 orOnce a new compound has been identified in evaluate its initial safety and tolerability profile, BLA, the new medicine becomes availablethe laboratory, medicines are usually devel- determine a safe dosage range, and identify for physicians to prescribe. A company mustoped 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 andpany conducts laboratory and animal studies to volunteer patients, usually between 100 and appropriate quality-control records. For someto show biological activity of the compound 300, to see if it iseffective, identify an optimal medicines, FDA requires additional trialsagainst 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 effectiveInvestigational New Drug Application (IND). Clinical Trials, Phase III—The drug is given to new medicines is a long, difficult, and expensiveAfter completing preclinical testing, a company a larger, more diverse patient population, often process. PhRMA member companies investedfiles 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 andAdministration (FDA) to begin to test the drug sometime many more thousands), to generate development in 2011.