FibroGen Receives Rare Pediatric Disease Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy
SAN FRANCISCO, April 15, 2021 (GLOBE NEWSWIRE) -- FibroGen, Inc., (NASDAQ: FGEN) announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD). Pamrevlumab has also received Fast Track designation from the U.S. Food and Drug Administration and is currently being evaluated in two Phase 3 trials for the treatment of DMD.
“Pediatric patients living with DMD face a significant unmet need with limited treatment options,” said Mark Eisner, M.D, M.P.H, Chief Medical Officer, FibroGen. “Obtaining Rare Pediatric Disease Designation is another acknowledgement of the serious and life threatening manifestations of this rare disease, and supports our mission to provide pamrevlumab as a potential new treatment option for patients suffering from DMD.”
About Rare Pediatric Disease Designation
Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics Licensing Application (BLA) for pamrevlumab for the treatment of DMD is approved by the FDA, FibroGen may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed to receive a priority review for any subsequent marketing application, or may be sold or transferred. This program is intended to encourage the development of new drugs and biologics for the treatment of rare pediatric diseases.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare and debilitating neuromuscular disease that affects approximately 1 in every 5,000 newborn boys. About 20,000 children are diagnosed with DMD globally each year. The fatal disease is caused by a genetic mutation leading to the absence or defect of dystrophin, a protein necessary for normal muscle function. The absence of dystrophin results in muscle weakness, muscle loss, fibrosis, and inflammation. Patients with DMD are often wheelchair-bound before the age of 12, and their progressive muscle weakness may lead to serious medical problems relating to respiratory and cardiac muscle.
Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.
FibroGen, Inc. is a biopharmaceutical company committed to discovering, developing, and commercializing a pipeline of first-in-class therapeutics. The Company applies its pioneering expertise in hypoxia-inducible factor (HIF) and connective tissue growth factor (CTGF) biology to advance innovative medicines for the treatment of unmet needs. The Company is currently developing and commercializing roxadustat, an oral small molecule inhibitor of HIF prolyl hydroxylase activity, for anemia associated with chronic kidney disease (CKD). Roxadustat is also in clinical development for anemia associated with myelodysplastic syndromes (MDS) and for chemotherapy-induced anemia (CIA). Pamrevlumab, an anti-CTGF human monoclonal antibody, is in clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For more information, please visit www.fibrogen.com.
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Michael Tung, M.D.
Corporate Strategy / Investor Relations