Drug-development challenges for small biopharmaceutical companies

New England Journal of Medicine

Volumn 376, Issue 5, 2017, Pages 469-474

  Moscicki, Richard A ^a   Tandon, P K ^b,c  

^a CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^b GENZYME CORPORATION   (United States)

^c ULTRAGENYX PHARMACEUTICAL INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALBIGLUTIDE; ALPHA GLUCOSIDASE; AMFEBUTAMONE PLUS NALTREXONE; ARIPIPRAZOLE LAUROXIL; BELINOSTAT; CANGRELOR; CHOLIC ACID; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DALBAVANCIN; DEOXYCHOLIC ACID; ELOSULFASE ALFA; ELUXADOLINE; GLOBOTRIAOSYLCERAMIDE; IVACAFTOR; MILTEFOSINE; PATIROMER; PERAMIVIR; PIRFENIDONE; PLACEBO; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; ROLAPITANT; SEBELIPASE ALFA; TAVABOROLE; URIDINE TRIACETATE; BIOLOGICAL MARKER; GENETIC MARKER;

ACIDURIA; AMINO ACID SUBSTITUTION; BILE ACID SYNTHESIS DISORDER; BILE DUCT DISEASE; CHEMOTHERAPY INDUCED NAUSEA; CYSTIC FIBROSIS; DIARRHEA; DRUG APPROVAL; DRUG DEVELOPMENT; DRUG INDUCED DISEASE; DRUG INDUSTRY; DRUG RESPONSE; ENZYME DEFICIENCY; ENZYME REPLACEMENT; FIBROSING ALVEOLITIS; FORCED EXPIRATORY VOLUME; GENETIC MARKER; GLYCOGEN STORAGE DISEASE TYPE 2; HEREDITARY OROTIC ACIDURIA; HUMAN; HYPERKALEMIA; INFLUENZA; IRRITABLE COLON; LEISHMANIASIS; LYSOSOMAL ACID LIPASE DEFICIENCY; MORQUIO A SYNDROME; MORQUIO SYNDROME; MUTATIONAL ANALYSIS; MYCOSIS; NON INSULIN DEPENDENT DIABETES MELLITUS; NONHUMAN; OBESITY; OVERALL SURVIVAL; PATIENT SELECTION; PERIPHERAL T CELL LYMPHOMA; PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTIVE VALUE; REVIEW; SAMPLE SIZE; SCHIZOPHRENIA; SKIN INFECTION; SMALL BIOPHARMACEUTICAL COMPANY; STUDY DESIGN; SURVIVAL RATE; SURVIVAL TIME; CLINICAL TRIAL (TOPIC); COMMERCIAL PHENOMENA; ECONOMICS; EPIDEMIOLOGY; KAPLAN MEIER METHOD; PLACEBO EFFECT; STANDARDS;

BIOMARKERS; CLINICAL TRIALS AS TOPIC; COMMERCE; DRUG DISCOVERY; DRUG INDUSTRY; GENETIC MARKERS; HISTORICALLY CONTROLLED STUDY; HUMANS; KAPLAN-MEIER ESTIMATE; PLACEBO EFFECT;

EID: 85011878976     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMra1510070     Document Type: Review

References (15)

1. Thomas D, Wessel C. Emerging therapeutic company investment and deal trends. Washington, DC: Biotechnology Industry Organization, 2015 (https://www.bio.org/sites/default/files/BIO%20Emerging%20Therapeutic%20Company%20Report%20June%2011%202015.pdf).
2. Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol 2014; 32: 40-51.
3. Kishnani PS, Hwu WL, Mandel H, et al. A retrospective, multinational, multicenter study on the natural history of infantileonset Pompe disease. J Pediatr 2006; 148: 671-6.
4. Kishnani PS, Nicolino M, Voit T, et al. Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease. J Pediatr 2006; 149: 89-97.
5. Kishnani PS, Corzo D, Nicolino M, et al. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology 2007;68:99-109.
6. Eng CM, Banikazemi M, Gordon RE, et al. A phase 1/2 clinical trial of enzyme replacement in Fabry disease: pharmaco-kinetic, substrate clearance, and safety studies. Am J Hum Genet 2001; 68: 711-22.
7. Eng CM, Guffon N, Wilcox WR, et al. Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry's disease. N Engl J Med 2001; 345:9-16.
8. Thurberg BL, Rennke H, Colvin RB, et al. Globotriaosylceramide accumulation in the Fabry kidney is cleared from multiple cell types after enzyme replacement therapy. Kidney Int 2002; 62: 1933-46.
9. Accurso FJ, Rowe SM, Clancy JP, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med 2010; 363: 1991-2003.
10. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663-72.
11. Barrett PM, Alagely A, Topol EJ. Cystic fibrosis in an era of genomically guided therapy. Hum Mol Genet 2012; 21: R1: R66-71.
12. Olanow CW, Kordower JH, Freeman TB. Fetal nigral transplantation as a therapy for Parkinson's disease. Trends Neurosci 1996;19:102-9.
13. Schumacher JM, Ellias SA, Palmer EP, et al. Transplantation of embryonic porcine mesencephalic tissue in patients with PD. Neurology 2000; 54: 1042-50.
14. Hauser RA, Watts RL, Freeman TB, et al. A double-blind, randomized, controlled, multicenter clinical trial of the safety and efficacy of transplanted fetal porcine ventral mesencephalic cells versus imitation surgery in patients with Parkinson's disease. Mov Disord 2001; 16: 983-4.
15. Goetz CG, Wuu J, McDermott MP, et al. Placebo response in Parkinson's disease: comparisons among 11 trials covering medical and surgical interventions. Mov Disord 2008;23:690-9.