Specialty Pharma Journal Specialty, Precision & Technology
SEATTLE–(BUSINESS WIRE)–Immusoft Corporation, a Seattle-Wash.-based cell therapy company, announced today that the U.S. Food and Drug Administration (FDA) has granted it Rare Pediatric Disease Designation (RPDD) for Iduronicrin genleukocel-T, Immusoft’s Sleeping Beauty transposon-engineered autologous plasmablasts for the expression and delivery of alpha-L-iduronidase (IDUA) to treat Mucopolysaccharidosis type I (MPS I). …
Tag Archives: Rare Pediatric Disease Designation
October, 2018
September, 2018
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19 September
FDA Grants Rare Pediatric Disease Designation to Cellectar’s Investigational Treatment for Osteosarcoma
MADISON, Wis., Sept. 17, 2018 (GLOBE NEWSWIRE) — Cellectar Biosciences, Inc. (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, announces today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR …
August, 2018
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23 August
FDA Grants Rare Pediatric Disease Designation to Krystal’s KB105 for Patients with TGM-1-Deficient Autosomal Recessive Congenital Ichthyosis
PITTSBURGH, Aug. 23, 2018 (GLOBE NEWSWIRE) — Krystal Biotech, Inc. (Nasdaq: KRYS), a gene therapy company dedicated to developing and commercializing novel treatments for patients suffering from dermatological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation (“RPDD”) to the company’s gene …
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16 August
Wave Life Sciences’ WVE-210201 Granted Orphan Drug and Rare Pediatric Disease Designations from the FDA for Treatment of DMD
CAMBRIDGE, Mass., Aug. 16, 2018 (GLOBE NEWSWIRE) — Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for …
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14 August
Cellectar’s CLR 131 Receives FDA Rare Pediatric Disease Designation for the Treatment of Ewing’s Sarcoma
MADISON, Wis., Aug. 13, 2018 (GLOBE NEWSWIRE) — Cellectar Biosciences (Nasdaq: CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, announces today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to CLR 131, …
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9 August
FDA Grants Prometic’s Rare Pediatric Disease Designation for its Alström Syndrome Drug
LAVAL, QC, Aug. 7, 2018 /PRNewswire/ – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“Prometic”) today announced that the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation to its small molecule drug candidate, PBI-4050, for the treatment of Alström syndrome (AS). In addition to …
May, 2018
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17 May
Myonexus Receives FDA Rare Pediatric Drug Designation for Investigational Treatment of Limb Girdle Muscular Dystrophy Type 2E
NEW ALBANY, Ohio–(BUSINESS WIRE)–Myonexus Therapeutics, a clinical-stage gene therapy company developing first ever corrective gene therapies for limb girdle muscular dystrophies, and Nationwide Children’s Hospital announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for the MYO-101 program, an AAV-based gene therapy for …
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3 May
Cellectar Receives Rare Pediatric Disease Designation for CLR 131 to Treat Neuroblastoma
MADISON, Wis., May 02, 2018 (GLOBE NEWSWIRE) — Cellectar Biosciences (Nasdaq:CLRB), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, announces today that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to the company’s lead …
January, 2018
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19 January
Orphazyme’s Arimoclomol Receives FDA Rare Pediatric Disease Designation for Niemann-Pick disease Type C
Copenhagen, Denmark, January 19, 2018 – Orphazyme A/S, a Danish biotech company listed on Nasdaq Copenhagen (TICKER: ORPHA.CO), with a late-stage, orphan-drug pipeline, today announced that arimoclomol has been granted rare pediatric disease designation by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease Type C (NPC). …
December, 2017
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8 December
MeiraGTx’s Leber’s Congenital Amaurosis Drug Receives Rare Pediatric Disease Designation from the FDA
LONDON and NEW YORK, Dec. 7, 2017 /PRNewswire/ — MeiraGTx, a London and New York-based gene therapy company, today announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted rare pediatric disease designation to the Company’s gene therapy product …