Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass.–(BUSINESS WIRE)–Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML). SY-1425, an oral first-in-class …
Tag Archives: Orphan Drugs
August, 2017
July, 2017
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20 July
FDA Grants Orphan Drug Designation to Astellas’ Gilteritinib for Treatment of Acute Myeloid Leukemia
TOKYO, July 20, 2017 /PRNewswire/ — Astellas Pharma Inc. (TSE: 4503, President and CEO: Yoshihiko Hatanaka, “Astellas”) announced today that the U.S. Food and Drug Administration (FDA) granted orphan-drug designation to gilteritinib in patients with acute myeloid leukemia (AML). The Orphan Drug Designation program assigns status to drugs and biologics …
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5 July
FDA Grants Orphan Drug Designation to Sobi’s Treatment for MPS IIIA
STOCKHOLM–(BUSINESS WIRE)–Swedish Orphan Biovitrum AB (publ) (Sobi™) has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the company’s development candidate SOBI003, a chemically modified human recombinant sulfamidase for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, a …
June, 2017
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29 June
FDA Unveils Plan to Eliminate Orphan Designation Backlog
Today, the U.S. Food and Drug Administration unveiled a strategic plan to eliminate the agency’s existing orphan designation request backlog and ensure continued timely response to all new requests for designation with firm deadlines. The agency’s Orphan Drug Modernization Plan comes a week after FDA Commissioner Scott Gottlieb committed to …
December, 2016
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2 December
AbbVie’s Risankizumab Receives Orphan Drug Designation for Treatment of Pediatric Patients with Crohn’s Disease
NORTH CHICAGO, Ill., Nov. 30, 2016 /PRNewswire/ — AbbVie (NYSE: ABBV), a global biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to risankizumab (ABBV-066; formerly BI 655066) for the investigational treatment of Crohn’s disease in pediatric patients. Risankizumab is being evaluated in …
November, 2016
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28 November
FDA Grants Orphan Drug Designation to PhaseRx’s Rare Liver Disorder Drug
SEATTLE, Nov. 28, 2016 /PRNewswire/ — PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in children, today announced that its lead candidate, PRX-OTC, which is being developed for the treatment of ornithine transcarbamylase deficiency (OTCD), has received orphan drug designation by the U.S. …
October, 2016
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19 October
FDA Awards 21 Grants to Stimulate Product Development for Rare Diseases
The U.S. Food and Drug Administration today announced that it has awarded 21 new clinical trial research grants totaling more than $23 million over the next four years to boost the development of products for patients with rare diseases. These new grants were awarded to principal investigators from academia and …
August, 2016
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19 August
Zymeworks Receives Orphan Status from the FDA for Two of its Ovarian Cancer Drugs
VANCOUVER, British Columbia–(BUSINESS WIRE)–Zymeworks Inc., a biopharmaceutical company discovering and developing innovative multi-functional protein-based therapeutics, including bi-specific antibodies and drug conjugates for the treatment of cancer, announced today that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Zymeworks’ lead investigational products ZW25 and ZW33 …
February, 2016
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25 February
AstraZeneca and Acerta Pharma’s Acalabrutinib Recommended for EU Orphan Status for 3 Indications
AstraZeneca and Acerta Pharma BV, a company in which AstraZeneca has a majority equity investment, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) adopted three positive opinions recommending acalabrutinib (ACP-196) for designation as an orphan medicinal product. The three positive opinions are for the …
May, 2015
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28 May
Cerulean Pharma’s Ovarian Cancer Drug Granted Orphan Drug Status
The US Food and Drug Administration (FDA) has granted Orphan Drug designation to Cerulean Pharma Inc.’s investigational ovarian cancer drug. The company announced that the agency granted the designation to its nanoparticle-drug conjugate (NDC), CRLX101, for the treatment of patients with ovarian cancer. The designation provides certain incentives, such as …