FDA Expands Use of Boehringer’s Therapy for Rare Lung Disease

The U.S. Food and Drug Administration approved the expansion of Boehringer Ingelheim’s Ofev as the first treatment for people with chronic fibrosing interstitial lung diseases with a progressive phenotype.

Ofev is currently approved for idiopathic pulmonary fibrosis and to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated fibrosis, two types of interstitial lung diseases.

Interstitial lung diseases (ILDs) include more than 200 disorders, which are classified as rare diseases. They can lead to pulmonary fibrosis – an irreversible scarring of lung tissue that negatively impacts lung function. Chronic fibrosing ILDs in which lung fibrosis continues to worsen are estimated to occur in 18 to 32 percent of patients with ILDs.

Ofev is a multi-targeted tyrosine kinase inhibitor that inhibits key pathways involved in lung fibrosis in ILDs and can slow the rate of decline in pulmonary function in patients. Ofev received Breakthrough Therapy designation from the FDA for chronic fibrosing ILDs with a progressive phenotype.

The FDA approval is based on the INBUILD trial, the first phase 3 clinical trial in the field of ILDs to group patients based on the clinical behavior of their disease rather than the primary clinical diagnosis. The study showed Ofev slowed lung function decline by 57 percent relative to placebo across overall study population as assessed by the annual rate of decline in forced vital capacity.

The phase 3 trial safety and tolerability profile of Ofev was consistent with what was previously seen in IPF studies. The most common adverse reactions reported in greater than or equal to 5 percent in Ofev-treated patients compared to placebo were diarrhea, nausea, abdominal pain, vomiting, liver enzyme elevation, decreased appetite, weight loss, headache, hypertension, nasopharyngitis, upper respiratory tract infection, urinary tract infections, fatigue, and back pain.

“Chronic fibrosing ILDs with a progressive phenotype lead to respiratory symptoms and worsening lung function,” said Kevin Flaherty, professor of medicine in the division of Pulmonary and Critical Care Medicine at the University of Michigan in Ann Arbor, Michigan, and lead investigator of the INBUILD trial. “This approval provides a therapeutic option for many patients who did not have an approved treatment until today.”

Photo: Kevin Flaherty, professor of medicine in the division of Pulmonary and Critical Care Medicine at the University of Michigan in Ann Arbor, Michigan, and lead investigator of the INBUILD trial

Author: Rare Daily Staff