Exhibit 99.1
FIBROGEN RECEIVES ORPHAN DRUG DESIGNATION FROM THE U.S. FDA FOR
PAMREVLUMAB FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
SAN FRANCISCO, California, April 15, 2019 – FibroGen, Inc. (NASDAQ: FGEN), a leading biopharmaceutical company discovering and developing a pipeline offirst-in-class therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD).
“We are pleased to have received Orphan Drug Designation from the FDA for pamrevlumab in the treatment of DMD. There is high unmet medical need for patients suffering from this debilitating disease needing a new treatment option,” said Elias Kouchakji, M.D., Senior Vice President, Clinical Development and Drug Safety. “All 21non-ambulatory DMD patients in our ongoing phase 2 study with pamrevlumab have completed the first 52 weeks of treatment. We are evaluating a number of clinical parameters in this study, including lung function, cardiac function, and upper extremity muscle function, and tissue fibrosis. We look forward to the continued development of this investigational therapeutic.”
Duchenne muscular dystrophy is a rare and debilitating neuromuscular disease that affects between approximately 1 in every 3,500 to 5,000 newborn boys. About 20,000 children are diagnosed with DMD globally each year. Progression tonon-ambulatory status typically occurs between the ages of 6 and 13 years. Once becomingnon-ambulatory, joint contractures and scoliosis develop rapidly, and may lead to cardiomyopathy and respiratory failure with fatality in young adulthood. While most DMD drugs in development target ambulatory DMD patients, the majority of DMD patients arenon-ambulatory. Pamrevlumab’s clinical program currently targets the unmet medical need in these patients.
About Orphan Drug Designation
Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. This designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing, to advance the evaluation and development of products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions. Orphan Drug Designation can also convey up to seven years of marketing exclusivity if the compound receives regulatory approval from the FDA.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare and debilitating neuromuscular disease that affects between approximately 1 in every 3,500 to 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.
