Novel Treatment For Progressive Pulmonary Fibrosis Gets Breakthrough Therapy Designation From the FDA

The U.S. Food and Drug Administration (FDA) has granted a novel antifibrotic Breakthrough Therapy designation. This move could speed up the therapy’s development for the treatment of people with progressive pulmonary fibrosis (PPF).

Pulmonary Fibrosis

The oral lysophosphatidic acid receptor 1 (LPA1) antagonist by Bristol Myers Squibb (BMS), named BMS-986278, has shown great potential as a therapy for patients with PPF. Its Breakthrough Therapy designation follows impressive results from a Phase 2 randomized, controlled trial that was reported at the European Respiratory Society (ERS) International Congress in September.

PPF is a chronic, potentially fatal interstitial lung disease (ILD) characterized by lung tissue scarring and damage. It exhibits a similar prognosis as idiopathic pulmonary fibrosis (IPF), which has a median, post-diagnosis survival time of three to five years.

The new designation for the investigational antifibrotic seems like a glimmer of hope for the hundreds of thousands of patients battling with PPF.

What is a Breakthrough Therapy designation?

Breakthrough Therapy is a program of the FDA aimed at speeding up both the development and regulatory review of medicines. The designation is usually granted to therapies for severe or life-threatening conditions. However, preliminary clinical evidence must exist to show the potential of such treatments.

With declining lung function, people with PPF are faced with unpleasant respiratory symptoms. They also have to contend with poor quality of life. These could eventually lead to respiratory failure and death.

“The FDA’s breakthrough therapy designation underscores the potential of BMS-986278 as an innovative, first-in-class treatment that may redefine the standard of care for progressive pulmonary fibrosis,” stated Roland Chen, MD, BMS’s senior vice president and head of immunology, cardiovascular and neuroscience development.

Trial backing

The Breakthrough Therapy designation is buoyed by data from a Phase 2 global, randomized study. The novel treatment was assessed in patients with IPF and PPF with great outcomes.

Trial subjects were randomized to get BMS-986278 (30 mg or 60 mg) or equal placebo orally two times per day. Researchers permitted patients in the PPF cohort to use background antifibrotics and/or immunosuppressants.

Percent-predicted FVC decline was found to be greater among patients placed on placebo at -4.3 percent. The rates were -2.7 percent and -1.1 percent among patients who received 30mg and 60mg BMS-986278 respectively.

Twice-daily treatment with 60mg BMS-986278 led to a 69 percent relative reduction in the rate of decline percent-predicted FVC, compared with placebo.

Interestingly, the treatment effect was observed to be consistent regardless of background therapy. The novel antifibrotic was also safe and well-tolerated while having only “very few treatment discontinuations.”

As development continues, BMS is planning to carry out a Phase 3 trial to further assess the efficacy and safety of the novel treatment in PPF patients.