Orphan Drug Designation

Soligenix, Inc. (NASDAQ:SNGX) stock is higher on Monday after the U.S. Food and Drug Administration granted orphan drug designation to dusquetide, the active ingredient in SGX945, for Behçet’s Disease. Follow SNGX stock’s rise today here. The update follows the review of recent ...

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Kymera Therapeutics (NASDAQ: KYMR) lead drug candidate KT-333 for the treatment of patients with Peripheral T-cell Lymphoma (PTCL). The company said that KT-333 is a first-in-class degrader of the...

The FDA has granted Orphan Drug Designation to Lantern Pharma Inc's (NASDAQ: LTRN) LP-184 for glioblastoma multiforme (GBM) and other malignant gliomas. The news follows the recent announcement of the FDA granting LP-184 Orphan Drug tag for pancreatic cancer. LP-184 is a small molecule drug...

The European Commission has granted Orphan Drug designation to Taysha Gene Therapies Inc's (NASDAQ: TSHA) TSHA-105, AAV9-based gene therapy for SLC13A5-related epilepsy. Related: Taysha Gene Secures $100M In Debt Financing For Support Pipeline Development SLC13A5...

The FDA has granted Orphan Drug Designation to Paratek Pharmaceuticals Inc's (NASDAQ: PRTK), Nuzyra (omadacycline) for infections caused by Nontuberculous Mycobacteria (NTM).  The orphan drug designation includes NTM pulmonary disease caused by Mycobacterium...

The FDA has granted Orphan Drug Designation to Allogene Therapeutics Inc's (NASDAQ: ALLO) ALLO-715 to treat multiple myeloma. In April 2021, ALLO-715 received Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA. ALLO-715, an AlloCAR T therapy targeting B-...

The FDA has granted Orphan Drug Designation to C4 Therapeutics Inc (NASDAQ: CCCC) CFT7455 for multiple myeloma. CFT7455 is an orally bioavailable MonoDAC degrader targeting IKZF1/3.  In June, C4 Therapeutics initiated a Phase 1/2 trial for CFT7455 to primarily investigate...

The FDA has granted Orphan Drug Designation to Lantern Pharma Inc's (NASDAQ: LTRN) LP-184 for pancreatic cancer.  LP-184 is a small molecule drug candidate and next-generation alkylating agent that preferentially damages DNA in cancer cells that over-express certain...

The European Medicines Agency (EMA) Committee for Orphan Medicinal Products has issued a positive opinion on Akouos Inc's (NASDAQ: AKUS) application for orphan drug designation for AK-OTOF. AK-OTOF is a gene therapy intended for the treatment of otoferlin gene-mediated...

The FDA has granted orphan drug designation to Aldeyra Therapeutics Inc's (NASDAQ: ALDX) ADX-2191 to treat retinitis pigmentosa (RP). ADX-2191 is a methotrexate for intravitreal injection. Methotrexate inhibits dihydrofolic reductase, an enzyme involved in cellular...

The FDA has granted Orphan Drug Designation to Relief Therapeutics Holding AG's (OTC: RLFTF) for RLF-100 (aviptadil), an inhaled formulation, for sarcoidosis. RLF-100 is a synthetic form of vasoactive intestinal peptide. Sarcoidosis is a rare disease in which the...

The FDA has granted Orphan Drug Designation to Novartis AG's (NYSE: NVS) NIS793 in combination with standard of care chemotherapy to treat pancreatic cancer. NIS793 is a fully human anti-TGF-β IgG2 monoclonal antibody designed to inhibit the TGF-β pathway in tumor...

The FDA has granted Orphan Drug Designation to Aldeyra Therapeutics Inc's (NASDAQ: ALDX) ADX-2191 (methotrexate for intravitreal injection) for primary vitreoretinal lymphoma (PVRL).  PVRL is a rare ocular lymphoid malignancy consisting of a subset of primary central...

The FDA has granted Orphan Drug Designation to Soleno Therapeutics Inc (NASDAQ: SLNO) DCCR (diazoxide choline) Extended-Release tablets for the treatment of Glycogen Storage Disease Type 1a (GSD 1a), or von Gierke disease. Von Gierke disease is a condition in which the body...

Seelos Therapeutics Inc's (NASDAQ: SEEL) Trehalose (SLS-005) has received Orphan Drug Designation for amyotrophic lateral sclerosis (ALS) from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Under the orphan designation, Seelos stands to...