Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral therapy, BBP-418, to treat limb-girdle muscular dystrophy type 2i (LGMD2i).
If approved, BBP-418 would become the first available treatment for LGMD2i, also called LGMDR9. In fact, according to Bridgebio, BBP-418 has the potential to become the first FDA-approved treatment for any form of LGMD, a category that encompasses several forms of muscular dystrophy marked by weakness and wasting of hip and shoulder muscles.
“This [new drug application] submission brings us one step closer to delivering the first approved therapy to individuals and families affected by LGMD2I/R9, a severe, progressive neuromuscular disease,” Christine Siu, CEO of Bridgebio Neuromuscular, said in a company press release. “This achievement not only reflects the strength of the data, but also our dedicated focus on addressing the urgency of a community that has long been waiting for meaningful treatment options. We are committed to working closely with the FDA to make this potential disease-modifying therapy available as quickly as possible.”
Bridgebio said that, based on its discussions with the FDA, it expects that BBP-418 will be approved and become commercially available later this year or early next year. The company is also engaging with regulatory agencies to identify an accelerated approval pathway for the therapy in Europe.
Aiding muscle function by fixing sugar problems
LGMD2i is caused by mutations that lead to problems with glycosylation, a biochemical process that involves the attachment of sugar molecules, in a muscle protein called alpha-dystroglycan. When this protein lacks the correct sugar molecules, it doesn’t function properly, leading to muscle damage that drives disease symptoms.
BBP-418 works by providing the body with more raw materials to attach these sugar molecules to the protein, boosting alpha-dystroglycan glycosylation and ultimately helping to preserve or improve muscle function.
Bridgebio’s application requesting FDA approval of BBP-418 was based primarily on data from a Phase 3 clinical trial called FORTIFY (NCT05775848), which tested the therapy against a placebo in people with LGMD2i, ages 12 to 60.
Bridgebio announced last year that the study had hit all its main goals. Results after three months showed that alpha-dystroglycan glycosylation levels nearly doubled in participants given BBP-418. After one year, patients given the experimental treatment tended to see improvements in measures of walking ability and lung function, whereas these measures generally worsened over time in patients on placebo.
The FDA previously granted BBP-418 orphan drug, rare pediatric disease, and fast track designations. These designations offer the possibility of accelerated review of the therapy’s application, and if BBP-418 is ultimately approved, the company will get access to several regulatory perks.
Bridgebio also said it plans to start testing BBP-418 in patients with LGMD2i younger than 12, as well as in people with another form of LGMD, called LGMD type 2m, in the near future.
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