FibroGen Receives Rare Pediatric Disease Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy Nasdaq:FGEN
SAN FRANCISCO, April 15, 2021 (GLOBE NEWSWIRE) – FibroGen, Inc. (NASDAQ: FGEN) announced that the U.S. Food and Drug Administration (FDA) has awarded the 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 studies for the treatment of DMD.
“Pediatric patients with DMD face a significant unmet need with limited treatment options,” said Mark Eisner, MD, MPH, Chief Medical Officer, FibroGen. “Obtaining the designation for rare pediatric diseases is another recognition of the serious and life-threatening manifestations of this rare disease, and supports our mission to offer pamrevlumab as a potential new treatment option for patients suffering from DMD.”
About the designation of rare pediatric conditions
Rare Pediatric Disease (RPD) Designation is granted by the FDA for serious or life-threatening illnesses that affect less than 200,000 people in the United States and where the serious or life-threatening manifestations primarily affect those under the age of 18. If a Biologics Licensing Application (BLA) for pamrevlumab for the treatment of DMD has been 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 each next marketing request, or can be sold or transferred. This program is designed to stimulate 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 about 1 in 5,000 newborn boys. About 20,000 children worldwide are diagnosed with DMD every year. The deadly disease is caused by a genetic mutation that results in the absence or defect of dystrophin, a protein required for normal muscle function. The absence of dystrophin leads to muscle weakness, muscle loss, fibrosis and inflammation. Patients with DMD are often wheelchair bound before the age of 12 and their progressive muscle weakness can lead to serious medical problems involving the respiratory and heart muscle.
Pamrevlumab is a premium 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 inoperable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD) and idiopathic pulmonary fibrosis (IPF). For information on pamrevlumab studies currently recruiting patients, visit www.clinicaltrials.gov.
FibroGen, Inc. is a biopharmaceutical company dedicated to discovering, developing and commercializing a pipeline of world-class therapies. The company applies its breakthrough expertise in hypoxia-inducible factors (HIF) and connective tissue growth factor (CTGF) biology to develop innovative drugs 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, a human monoclonal antibody against CTGF, is in clinical development for the treatment of locally advanced inoperable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD) and idiopathic pulmonary fibrosis (IPF). For more information, please visit www.fibrogen.com.
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From FibroGen, Inc.
Michael Tung, MD
Business strategy / investor relations