Celularity Seeks FDA Orphan Drug Status for PDA-002 to Treat FSHD

Celularity Inc., a company at the forefront of regenerative medicine, has submitted an application to the U.S. Food and Drug Administration (FDA) for orphan drug designation for PDA-002, its placental-derived cell therapy. The therapy is designed to treat Facioscapulohumeral Muscular Dystrophy (FSHD), a debilitating condition that leads to progressive muscle atrophy and affects approximately one in 8,000 individuals. Currently, there are no curative or disease-modifying treatments available for FSHD.
The orphan drug designation is granted to drugs that demonstrate potential in treating serious or life-threatening diseases that impact less than 200,000 people in the United States. Celularity has already received clearance from the FDA for an Investigational New Drug Application for PDA-002 and is planning to start a Phase 1/2 clinical study in the latter half of 2024. The results of this trial will be crucial for a future Biologics License Application (BLA) submission to the FDA, aiming to establish PDA-002 as a potential treatment for FSHD and other muscular dystrophies.
Celularity's CEO, Chairman, and Founder, Robert Hariri, M.D., Ph.D., highlighted the company's dedication to developing superior therapies for patients with limited treatment options. With the FDA's IND clearance, Celularity aims to utilize its cell therapy platform to enhance outcomes for FSHD patients and potentially address other neurodegenerative disorders. The company's extensive cell therapy portfolio encompasses T-cells, natural killer (NK) cells, mesenchymal stem cells (MSCs), and exosomes.
Celularity is focused on creating allogeneic, cryopreserved, placental-derived cell therapies that target aging-related diseases, including cancer and immune disorders. The company also develops advanced biomaterial products derived from the postpartum placenta, believing in the therapeutic potential of placental biology and its availability to meet significant global health needs for effective and affordable treatments.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!