FDA Grants Rare Pediatric Disease Designation to FibroGen Treatment for DMD

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation for FibroGen’s pamrevlumab for the treatment of patients with Duchenne muscular dystrophy.

“Pediatric patients living with Duchenne muscular dystrophy(DMD) face a significant unmet need with limited treatment options,” said Mark Eisner, chief medical officer at 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.”

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, Duchenne muscular dystrophy, and idiopathic pulmonary fibrosis. Pamrevlumab has also received Fast Track designation from the FDA.

The FDA grants Rare Pediatric Disease designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the United States. The designation makes pamrevlumab eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Rhythm Pharmaceutics sold its priority review voucher for $100 million in January 2021 to Alexion Pharmaceuticals.

Photo: Mark Eisner, chief medical officer at FibroGen