FDA application seeks approval of inhaled therapy for PAH
Submission is supported by PROSERA, TORREY, and additional analyses
Written by |
Gossamer Bio has submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of seralutinib, an inhaled therapy for people with pulmonary arterial hypertension (PAH).
The application is supported by data from the Phase 3 PROSERA study (NCT05934526), confirmatory evidence from the Phase 2 TORREY study (NCT04456998), and additional supportive analyses. PROSERA’s primary endpoint, however, narrowly missed its prespecified statistical threshold.
Gossamer’s submission, announced in a company press release, followed a pre-New Drug Application (NDA) Type B meeting with the FDA in June. The agency characterized the degree of statistical significance and magnitude of the treatment effect seen in PROSERA as review issues rather than filing issues. The FDA also provided feedback on the format and content of the planned submission.
FDA to decide whether to accept seralutinib application
The FDA will decide whether to accept the application for filing after its initial review, which the company anticipates could occur later this year.
“Submitting our first [new drug application] is a significant milestone for Gossamer and, more importantly, for patients living with PAH,” said Faheem Hasnain, chairman, co-founder, and CEO of Gossamer.
In PAH, the arteries that carry blood through the lungs narrow, raising blood pressure and forcing the right side of the heart to work harder. Over time, this strain can lead to heart failure. Abnormal growth of cells in the walls of these blood vessels contributes to their narrowing.
Gossamer is developing seralutinib in collaboration with the Chiesi Group. The inhaled therapy is designed to block certain signaling proteins involved in the abnormal cell growth that contributes to PAH.
PROSERA enrolled 390 PAH patients, many of whom were heavily pretreated. Participants were randomly assigned to receive seralutinib or placebo twice daily by inhalation for about a year. The trial’s main goal was to assess the impact of seralutinib on the six-minute walk test (6MWT), a measure of exercise capacity, after about six months.
Compared with placebo, seralutinib was associated with a 13-meter (slightly longer than 40 feet) greater improvement in 6MWT distance. According to the trial’s prespecified criteria, this difference was not statistically significant, but Gossamer noted that it would have met a more commonly used threshold for statistical significance.
Higher-risk subgroup showed greater walking-distance benefit
In a prespecified subset of 234 patients who had intermediate- or high-risk disease at the study’s start, seralutinib was associated with a 20-meter (66-foot) greater improvement in 6MWT distance than placebo, a statistically significant difference. Most secondary endpoints also showed a statistically significant effect favoring seralutinib in this higher-risk group.
In a biomarker analysis, seralutinib reduced NT-proBNP levels in blood, a marker of heart damage, relative to placebo.
Safety findings from PROSERA indicated seralutinib was generally well tolerated, with overall rates of serious safety issues being comparable between the seralutinib and placebo groups. Even so, patients on seralutinib more often showed elevations in liver damage markers.
According to Gossamer, the improvements seen among higher-risk patients in PROSERA were consistent with the positive data from the earlier TORREY study.
“We are grateful to the patients, families, investigators and site staff who made PROSERA and TORREY possible, and to our employees for the work it took to reach this point,” Hasnain said.
Caryn Peterson, the company’s chief development officer, added that the FDA submission “brings us one step closer to delivering seralutinib as a potential new therapy for PAH patients who need better options.”
Gossamer paused enrollment in its Phase 3 SERANATA study (NCT07181382) of seralutinib in pulmonary hypertension associated with interstitial lung disease, a condition marked by scarring in the lungs. The company said the pause was pending further analysis of the PROSERA data, particularly because of regional differences in placebo responses.
Leave a comment
Fill in the required fields to post. Your email address will not be published.