The Food and Drug Administration (FDA) has granted Fast Track designation to pamrevlumab for the treatment of patients with Duchenne muscular dystrophy (DMD).
Pamrevlumab is a first-in-class fully human antibody that works by inhibiting the activity of connective tissue growth factor (CTGF), a common factor in fibrotic and proliferative disorders.
The designation is based on a review of a single-arm phase 2 trial (ClinicalTrials.gov: NCT02606136), which assessed the efficacy and safety of pamrevlumab in non-ambulatory patients 12 years of age and older with DMD. The Company is currently investigating pamrevlumab for the treatment of DMD in two phase 3 trials (ClinicalTrials.gov: NCT04632940, NCT04371666).
The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to drugs that are expected to have an impact on factors such as survival and daily functioning.
“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, MD, MPH, Chief Medical Officer, FibroGen. “We look forward to working closely with the FDA on the development of pamrevlumab as a potential therapy for DMD.”
The FDA previously granted Orphan Drug designation to pamrevlumab for this indication. The Company is also evaluating pamrevlumab for the treatment of idiopathic pulmonary fibrosis and locally advanced unresectable pancreatic cancer in phase 3 studies.
FibroGen receives Fast Track designation from the U.S. FDA for pamrevlumab for the treatment of Duchenne muscular dystrophy. [press release]. San Francisco, CA: FibroGen, Inc.; April 12, 2021.