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Celularity Submits Request to U.S. FDA for Orphan Drug Designation for PDA-002 Asset Treating Facioscapulohumeral Muscular Dystrophy

2024-03-21

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细胞疗法孤儿药临床研究免疫疗法临床申请

March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating Facioscapulohumeral Muscular Dystrophy (FSHD).

Orphan drug designation is a status given to drugs that show the potential to treat, prevent or diagnose serious or life-threatening diseases that affect fewer than 200,000 people in the U.S. Earlier, Celularity received FDA Investigational New Drug Application clearance for PDA-002 in FSHD and plans to commence a phase 1/2 study in the second half of 2024. The trial serves as an important component for submitting a Biologics License Application (BLA) to the FDA in the future as a potential treatment for FSHD and other types of muscular dystrophy.

Affecting approximately one in 8,000 people, FSHD is one of the most common types of muscular dystrophy, characterized by severe progressive, asymmetrical muscle atrophy that affects facial, upper and lower limb, and shoulder skeletal muscles. There is currently no cure or disease-modifying treatments for FSHD. The disease can cause significant lifetime morbidity, with approximately 24 percent of patients at risk of wheelchair use within six years of their diagnosis.

“Celularity is committed to developing best-in-class therapies for patients who suffer from challenging diseases who have few, if any, currently available treatment options,” said Robert Hariri, M.D., Ph.D., Celularity CEO, Chairman and Founder. “Having already received IND clearance from the FDA, we intend to leverage our cell therapy platform to improve outcomes for people living with FSHD and potentially other neurodegenerative disorders and believe these cellular immunotherapies have breakthrough potential. We look forward to commencing our Phase 1/2 study as we continue to push forward our extensive cell therapy portfolio, including T-cells, natural killer (NK) cells, mesenchymal stem cells (MSCs), and exosomes.”

About Celularity

Celularity Inc. (NASDAQ: CELU) is a cell therapy and regenerative medicine company developing allogeneic, cryopreserved, placental-derived cell therapies, including therapeutic programs using mesenchymal-like adherent stromal cells (MLASCs), T-cells engineered with CAR (CAR T-cells) and genetically modified and unmodified natural killer (NK) cells. These therapeutic programs target aging-related diseases, including degenerative diseases, cancer, and immune disorders. In addition, Celularity develops, manufactures, and commercializes advanced biomaterial products also derived from the postpartum placenta. Celularity believes that, by harnessing the placenta’s unique biology and ready availability, it can develop therapeutic solutions that address significant unmet global needs for effective, accessible and affordable therapies.

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