Rare Diseases

Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne Muscular Dystrophy and All Other Indications

Pratteln, Switzerland, and Rockville, MD, USA, September 2, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces that it has signed agreements with Idorsia (SIX: IDIA) and ReveraGen BioPharma Inc., making Santhera a direct license holder of vamorolone. Under the agreements, Santhera has obtained an exclusive license from ReveraGen, the originator of vamorolone, for …

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FDA Grants Mustang Bio’s MB-207 Rare Pediatric Disease Designation for Treatment of X-linked Severe Combined Immunodeficiency in Previously Transplanted Patients

WORCESTER, Mass., Aug. 31, 2020 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the U.S. Food and Drug Administration …

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FDA Grants Orphan Drug Designation for BCX9930 for Treatment of Paroxysmal Nocturnal Hemoglobinuria

RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation for its oral Factor D inhibitor, BCX9930, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). According to the FDA, the Orphan Drug Designation …

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Phase 2 Study of Soticlestat Meets Primary Endpoint Reducing Seizure Frequency in Children with Dravet Syndrome or Lennox-Gastaut Syndrome

NEW YORK & OSAKA, Japan–(BUSINESS WIRE)–Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) and Ovid Therapeutics Inc. (NASDAQ: OVID) (“Ovid”), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced positive topline results from the randomized Phase 2 ELEKTRA study of soticlestat in …

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FDA Accepts Sarepta’s NDA for Casimersen for Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 45

CAMBRIDGE, Mass., Aug. 25, 2020 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) seeking accelerated approval for casimersen (SRP-4045) and provided a regulatory action date …

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CENTOGENE and Evotec Expand Collaboration into Gaucher Disease

CAMBRIDGE, Mass. and ROSTOCK, Germany and HAMBURG, Germany and BERLIN, Aug. 20, 2020 (GLOBE NEWSWIRE) — Centogene N.V. (Nasdaq: CNTG), a commercial-stage company focused on rare diseases that transforms real-world clinical and genetic data into actionable information for patients, physicians, and pharmaceutical companies, and Evotec SE (Frankfurt Stock Exchange: EVT, …

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Polaryx Therapeutics Receives FDA Fast Track Designation for PLX-200 for the Treatment of Patients with Juvenile Neuronal Ceroid Lipofuscinosis

PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ — Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients. JNCL/CLN3 is …

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Mustang Bio’s MB-107 Granted Rare Pediatric Disease Designation from the FDA for Treatment of X-linked Severe Combined Immunodeficiency

WORCESTER, Mass., Aug. 17, 2020 (GLOBE NEWSWIRE) — Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced that the U.S. Food and Drug Administration …

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FDA Approves Genentech’s Enspryng for Treatment of Neuromyelitis Optica Spectrum Disorder

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Enspryng™ (satralizumab-mwge) as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD). NMOSD …

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Scholar Rock’s Investigational Spinal Muscular Atrophy Drug Receives Rare Pediatric Disease Designation from the FDA

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular …

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