FDA Grants Breakthrough Therapy Designation to Boehringer for Experimental IPF Therapeutic

The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Boehringer Ingelheim’s novel investigational therapy, BI 1015550, for the treatment of idiopathic pulmonary fibrosis.

Photo: Thomas Seck, senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals

BI 1015550 is an oral, phosphodiesterase 4B (PDE4B) inhibitor with the potential to address both pulmonary fibrosis – an irreversible scarring of lung tissue that negatively impacts lung function – and inflammation associated with progressive fibrosing interstitial lung diseases (ILDs). ILDs encompass more than 200 disorders that can lead to pulmonary fibrosis – an irreversible scarring of lung tissue that negatively impacts lung function and may become life-threatening.

Idiopathic pulmonary fibrosis (IPF) is one of the more common progressive fibrosing ILDs. Symptoms of IPF include breathlessness during activity, a dry and persistent cough, chest discomfort, fatigue, and weakness. Although IPF is a rare disease in the United States, it affects approximately 3 million people worldwide, primarily people over the age of 50 and more men than women.

“The accelerated development of BI 1015550 is part of Boehringer Ingelheim’s next wave of potential innovative treatments for interstitial lung diseases aimed at preserving lung function and improving the lives of patients,” said Thomas Seck, senior vice president, Medicine and Regulatory Affairs, Boehringer Ingelheim Pharmaceuticals. “BI 1015550 represents the first molecule in the class of PDE4B inhibitors that is being studied for IPF and other progressive fibrosing ILDs.” 

BI 1015550 was studied as a monotherapy or in combination with background antifibrotic therapy to assess the effectiveness of slowing the rate of lung function decline in patients with IPF. The efficacy, safety, and tolerability of BI 1015550 was studied in a Phase 2 randomized, double-blind, placebo-controlled trial of patients with IPF. The primary endpoint was change from baseline in Forced Vital Capacity (FVC) – a measure of lung function – over a 12-week treatment period. The results from the phase 2 study will be presented at the American Thoracic Society 2022 session in May.

The FDA Breakthrough Therapy designation is supported by data collected to date. Initiation of the phase 3 program is expected later this year.

The FDA established the Breakthrough Therapy designation process to expedite the development and review of drugs for serious or life-threatening conditions where preliminary clinical evidence indicate that the therapy may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints.

Author: Rare Daily Staff