Specialty Pharma Journal Specialty, Precision & Technology
CULVER CITY, Calif.–(BUSINESS WIRE)–NantKwest, Inc. (Nasdaq:NK), a pioneering, next generation, clinical-stage immunotherapy company focused on harnessing the unique power of our immune system using natural killer (NK) cells to treat cancer, infectious diseases and inflammatory diseases, announced today that the FDA has granted Orphan Drug Designation to the company’s activated …
Tag Archives: Orphan Drug
March, 2017
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17 March
Three Harvard Experts Explain how Economics Can Shape Precision Medicines
Author affiliations: Harvard Business School, Harvard Medical School, Dana-Farber/Brigham and Women’s Cancer Center, Harvard Kennedy School Many public and private efforts focus on research in precision medicine, the process by which genomic information and other characteristics of a patient’s disease are used to predict which treatments will be most effective. …
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7 March
Exelixis’ Hepatocellular Carcinoma Drug Granted Orphan Drug Designation from the FDA
SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Exelixis, Inc. (Nasdaq: EXEL) today announced that the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to cabozantinib for the treatment of hepatocellular carcinoma (HCC). This information was posted to FDA’s website on March 4, 2017 and can be accessed here. A pivotal …
November, 2016
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9 November
FDA Grants Orphan Drug Designation to Eloxx’s Enzyme Deficiency Drug
NESS ZIONA, Israel and WALTHAM, Massachusetts, November 7, 2016 /PRNewswire/ — Eloxx Pharmaceuticals, a biotechnology company focused on discovery, development and commercialization of compounds for the treatment of genetic diseases caused by nonsense mutations, announced today that the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) …
October, 2016
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24 October
Solid Biosciences’ Gene Therapy Granted Orphan Drug Status for Duchenne Muscular Dystrophy
CAMBRIDGE, Mass. & LONDON–(BUSINESS WIRE)–Solid Biosciences and its subsidiary, Solid GT, announced today that the U.S. Food and Drug Administration and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD). Solid plans to initiate …
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10 October
FDA Approves Lundbeck’s Carnexiv Injection as Intravenous Option for Patients with Certain Seizure Types
DEERFIELD, Ill.–(BUSINESS WIRE)–Lundbeck announced today that the U.S. Food and Drug Administration (FDA) has approved Carnexiv™ (carbamazepine) injection as a short-term replacement therapy for oral carbamazepine formulations in adults with certain seizure types when oral administration is temporarily not feasible. Carnexiv received orphan drug designation for this indication and will …
August, 2016
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15 August
FDA Grants Orphan Drug Designation to Tivorsan Pharmaceuticals’ Duchenne Muscular Dystrophy Drug
PROVIDENCE, R.I.–(BUSINESS WIRE)–Tivorsan Pharmaceuticals, Inc., a biotechnology company that is pioneering a novel approach to treating all forms of Duchenne Muscular Dystrophy (DMD) and other serious, debilitating neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead investigational drug, human …
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10 August
Spark Therapeutics Announces New Positive Long-Term Data from its Late-Stage Orphan Eye Drug Trial
PHILADELPHIA, Aug. 10, 2016 (GLOBE NEWSWIRE) — Spark Therapeutics (NASDAQ:ONCE) today announced new data from the continuation of the Phase 3 trial of voretigene neparvovec (formerly referred to as SPK-RPE65), its most advanced product candidate. Voretigene neparvovec, for the potential treatment of inherited retinal disease (IRD) caused by mutations in the …
May, 2016
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12 May
AstraZeneca’s Thyroid Cancer Drug Granted Orphan Drug Status from the FDA
AstraZeneca today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for the investigational MEK 1/2 inhibitor, selumetinib (AZD6244, ARRY-142886) for adjuvant treatment of patients with stage III or IV differentiated thyroid cancer (DTC). DTC is diagnosed in approximately 60,000 people in the US each …
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6 May
Allena’s Investigational Pediatric Hyperoxaluria Drug Receives Orphan Drug Designation from the FDA
NEWTON, Mass.–(BUSINESS WIRE)–Allena Pharmaceuticals, Inc., a specialty biopharmaceutical company focused on developing and commercializing innovative, non-systemic, oral protein therapeutics to treat metabolic and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Allena’s investigational therapy oxalate decarboxylase for the treatment of …