FDA Grants FibroGen Fast Track Designation for DMD Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted FibroGen Fast Track designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy.

The designation follows review of the phase 2 clinical data from a single-arm trial in non-ambulatory patients with Duchenne muscular dystrophy (DMD) and represents recognition by the FDA that pamrevlumab has the potential to address an unmet medical need for the disease. Pamrevlumab is currently being evaluated in two phase 3 trials for the treatment of DMD.

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).

Fast Track designation is intended to facilitate the development and review of drugs used to treat serious conditions and to fill an unmet medical need. Fast Track designation enables the company to have more frequent interactions with the FDA throughout the drug development process, so that an approved product can reach the market expeditiously.

“Fast Track designation by the FDA for pamrevlumab in DMD underscores the high unmet medical need for patients suffering from this debilitating disease and potential to advance a new treatment option,” said Mark Eisner, chief medical officer of FibroGen.

Photo: Mark Eisner, chief medical officer of FibroGen