Celularity Seeks FDA Orphan Status for PDA-002 in FSHD Treatment

New Jersey-based biotechnology firm Celularity Inc. has made a significant move in the field of regenerative medicine by submitting an application to the U.S. Food and Drug Administration (FDA) for orphan drug status for its innovative cell therapy, PDA-002. This therapy, derived from placental cells and readily available, is designed to treat Facioscapulohumeral Muscular Dystrophy (FSHD), a serious condition affecting roughly one in 8,000 people and characterized by progressive muscle degeneration.

FSHD is a form of muscular dystrophy that leads to asymmetrical muscle atrophy, impacting facial, upper and lower limb, and shoulder muscles. The disease is associated with significant morbidity and a lack of effective treatments or cure, with nearly a quarter of patients potentially requiring wheelchair use within six years of diagnosis.

Celularity's CEO, Robert Hariri, emphasized the company's dedication to developing superior therapies for patients with limited treatment options. The FDA has already granted Investigational New Drug Application clearance for PDA-002, paving the way for a phase 1/2 clinical study scheduled to begin in the latter half of 2024. This study is crucial for the development of a Biologics License Application for potential treatment of FSHD and other muscular dystrophies.

Celularity's focus is on developing allogeneic, cryopreserved, placental-derived cell therapies, including mesenchymal-like adherent stromal cells, CAR T-cells, and natural killer cells. These therapies aim to address aging-related diseases, cancer, and immune disorders. The company also produces advanced biomaterial products from the postpartum placenta, with the goal of creating accessible and affordable treatments to meet significant global health needs.

The orphan drug designation, if granted, would recognize the potential of PDA-002 to treat a serious condition affecting a small population in the U.S. This designation could provide Celularity with incentives to further the development of PDA-002 and bring a much-needed treatment to FSHD patients. The company's comprehensive cell therapy portfolio, including T-cells, NK cells, mesenchymal stem cells, and exosomes, demonstrates its commitment to advancing cellular immunotherapies with breakthrough potential.

Celularity's forward-looking approach is evident in its ongoing efforts to expand its cell therapy offerings and to contribute to the advancement of regenerative medicine. The company's mission is to leverage the unique biology of the placenta to develop therapeutic solutions that can make a significant impact on global health.