The FDA has awarded orphan drug designation to Papillon Therapeutics Inc.’s PPL-002, an experimental gene-modified CD34+ hematopoietic stem and progenitor cell (HSPC) therapy, for the treatment of Danon disease.
The FDA has awarded U.S. orphan drug designation to Eydisbio Inc.’s EYD-001 (formerly HS-276), a highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of systemic sclerosis. Eydisbio plans to initiate clinical trials in the near future.
Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy.
Somite Therapeutics Inc.’s lead program, SMT-M01, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Duchenne muscular dystrophy (DMD). The program leverages the company’s proprietary Alphastem artificial intelligence (AI) platform to develop a novel cell replacement therapy for DMD.
South Korean artificial intelligence-based drug developer Pharos Ibio Co. Ltd. said that the Ministry of Food and Drug Safety (MFDS) granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia (AML).
Abdera Therapeutics Inc.’s ABD-147 has been granted U.S. orphan drug designation for the treatment of neuroendocrine carcinoma. ABD-147 is a next-generation precision radiopharmaceutical biologic therapy designed to deliver actinium-225 (225Ac) to solid tumors expressing DLL3.
South Korean artificial intelligence-based drug developer Pharos Ibio Co. Ltd. said that the Ministry of Food and Drug Safety (MFDS) granted an orphan drug designation for PHI-101, a second-line therapy for acute myeloid leukemia (AML).
Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.
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