FDA to decide on potential first therapy for LGMD2i by late November
Bridgebio prepares to launch BBP-418 as soon as the treatment's approved
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No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may change later this year.
The U.S. Food and Drug Administration (FDA) has accepted an application from Bridgebio Pharma seeking approval for its experimental oral therapy BBP-418. The agency granted the application priority review, reducing the review period from the usual 10 months to about six months.
A final decision from the FDA is expected by Nov. 27. If approved, BBP-418 would become the first FDA-approved therapy for LGMD2i, which is also known as LGMDR9. Bridgebio said it is anticipating a positive decision and is ready to commercially launch the treatment as soon as the FDA gives the okay.
“LGMD2I/R9 is a relentless and life-shortening disease,” Christine Siu, CEO of Bridgebio Neuromuscular, said in a company press release. “Patients progressively lose the ability to walk, face serious cardiovascular complications, and ultimately die from respiratory failure. With today’s acceptance of our NDA [new drug application], we are one step closer to the potential FDA approval of a treatment that could potentially change the progression of this disease.”
Patient community reacts to the FDA decision
Advocates cheered the FDA’s decision to review Bridgebio’s application.
“For families living with LGMD2I/R9, every milestone reflects years of advocacy, resilience, and hope. … We are grateful for the continued commitment to patients and families who have waited far too long,” said Kat Bryant Knudson, founder and CEO of The Speak Foundation, a group that advocates for people with limb-girdle muscular dystrophy.
Bryant Knudson added that the FDA’s decision to review the application “reflects not only scientific advancement, but a commitment to listening to and partnering with our community every step of the way.”
LGMD2i is a genetic disease that causes problems with a muscle protein called alpha-dystroglycan. Normally, certain sugar molecules attach to alpha-dystroglycan via a biochemical process called glycosylation; these added sugar molecules are essential for the protein’s proper function. But in LGMD2i, glycosylation is impaired, disrupting the protein’s activity and ultimately driving disease symptoms.
BBP-418 is designed to boost glycosylation of the alpha-dystroglycan protein by providing the body with additional raw materials for attaching these sugar molecules. By increasing glycoslyation, the therapy aims to increase the protein’s function and ultimately preserve or improve muscle function.
Bridgebio’s application seeking FDA approval was based mainly on findings from the Phase 3 FORTIFY clinical trial (NCT05775848). That study tested BBP-418 against a placebo in approximately 80 people with LGMD2i, ages 12 to 60. Results showed benefits over placebo as early as three months into treatment in measures of walking. In addition, improvements were also noted in the increase in alpha-dystroglycan glycosylation levels, the decrease in a key marker of muscle damage, and in a measure of lung function.
“The compelling data from FORTIFY give us confidence that BBP-418 can make a meaningful difference in how this disease progresses, and we will work with urgency to bring it to the patients and families who have been waiting,” Siu said.
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