FibroGen (FGEN) Receives Rare Pediatric Disease Designation from the U.S. FDA for Pamrevlumab for Treatment of DMD
- Wall Street ends volatile week sharply higher
- Disney (DIS) Dips As Streaming Business Shows Signs of Slowing Down, Analysts 'Very Bullish' on LT Opportunity
- Airbnb (ABNB) Bookings Up 52% to Help Sales Beat, Seen as a 'Blow Out Quarter' as More Upside is Left
- DoorDash (DASH) Reports Bigger Loss and a Sales Beat, Shares Soars on Strong Guidance and Two Upgrades to 'Buy'
- Coinbase (COIN) To Add Dogecoin Trading in the Next Few Weeks
News and research before you hear about it on CNBC and others. Claim your 1-week free trial to StreetInsider Premium here.
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 DesignationRare 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.
About PamrevlumabPamrevlumab 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.
Serious News for Serious Traders! Try StreetInsider.com Premium Free!
You May Also Be Interested In
- BridgeBio Pharma (BBIO) Receives FDA Fast Track Designation for Investigational Gene Therapy for Congenital Adrenal Hyperplasia
- Aeterna Zentaris (AEZS) Begins Pivotal Phase 3 DETECT-Trial for Diagnosis of Childhood-Onset Growth Hormone Deficiency
- Pulse Biosciences (PLSE) Presents Clinical Study Results of Nano-Pulse Stimulation Technology to Clear Benign Lesions at ASLMS
Create E-mail Alert Related CategoriesCorporate News, FDA, Hot FDA News
Sign up for StreetInsider Free!
Receive full access to all new and archived articles, unlimited portfolio tracking, e-mail alerts, custom newswires and RSS feeds - and more!