FLORHAM PARK, N.J. - Celularity Inc. (NASDAQ: CELU), a biotechnology firm specializing in regenerative medicine, has formally requested orphan drug designation from the U.S. Food and Drug Administration (FDA) for PDA-002, its placental-derived cell therapy intended for the treatment of Facioscapulohumeral Muscular Dystrophy (FSHD). Orphan drug status is granted to treatments for rare diseases affecting fewer than 200,000 individuals in the United States and can provide certain benefits, including tax credits and market exclusivity.
The company previously secured FDA Investigational New Drug Application clearance for PDA-002 in FSHD and anticipates initiating a phase 1/2 study in the latter half of 2024. This clinical trial is a critical step towards a potential Biologics License Application submission for FSHD treatment, a debilitating condition with no current cure or disease-modifying therapies. FSHD, one of the most prevalent forms of muscular dystrophy, impacts around 1 in 8,000 people and leads to severe muscle wasting.
Celularity's CEO, Robert Hariri, M.D., Ph.D., emphasized the company's commitment to addressing the unmet needs of patients with challenging diseases through their cell therapy platform. The upcoming study is part of Celularity's broader strategy to develop cellular immunotherapies for neurodegenerative disorders.
The company's portfolio includes a range of cell therapies derived from the placenta, targeting diseases related to aging, cancer, and immune disorders. In addition to PDA-002, Celularity is working on mesenchymal stem cells (MSCs), T-cells, natural killer (NK) cells, and exosomes for therapeutic applications.
The information in this article is based on a press release statement from Celularity Inc.
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