BridgeBio: Opportunity for Accelerated Approval Pathway in Muscular Dystrophy Type

 BridgeBio met with the U.S. Food and Drug Administration (FDA) to discuss the use of glycosylated αDG levels as a surrogate endpoint; based on this meeting, the Company believes there is potential to pursue Accelerated Approval in the U.S. for BBP-418

- BridgeBio has dosed the first participant in FORTIFY, its global Phase 3 study of BBP-418 in patients with LGMD2I/R9

- FORTIFY includes an interim analysis at 12 months focused on change in glycosylated αDG levels; topline data from this analysis is expected in late 2024/early 2025

- Deficiency of glycosylated αDG is the causal molecular driver of LGMD2I/R9; in the ongoing Phase 2 study, patients treated with BBP-418 had a rapid and sustained increase of glycosylated αDG levels, concurrent with sustained decreases in creatine kinase and improvements from baseline in ambulatory and clinical function measures

- If successful, BBP-418 has the potential to address serious unmet need for patients with LGMD2I/R9, a disease for which there are no approved therapies

BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that, based on discussions with the U.S. Food and Drug Administration (FDA), there is a potential path to pursue accelerated approval in the U.S. for BBP-418, an investigational oral substrate supplementation therapy, in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9). BridgeBio also announced that the first patient with LGMD2I/R9 has been dosed in its Phase 3 FORTIFY clinical trial of BBP-418. To date, the Company has activated over half the planned U.S. clinical sites and is in the process of opening sites in Europe and Australia to support global registration.

The Phase 3 FORTIFY registrational study is a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of BBP-418. FORTIFY has a planned interim analysis at 12 months focused on assessing glycosylated αDG as a surrogate endpoint to potentially support an accelerated approval. The North Star Assessment for Dysferlinopathy (NSAD) and secondary endpoints will be evaluated at 36 months, and results are expected to provide confirmatory clinical data. A novel, validated bioassay was developed by BridgeBio to directly measure glycosylated ⍺DG levels, which are central to LGMD2I/R9 disease, and may enable monitoring of responses to disease-modifying therapies in LGMD2I/R9 patients. BridgeBio is committed to collaborating with the FDA to address the challenges associated with LGMD2I/R9 drug development, including the potential use of a surrogate endpoint to support an accelerated approval.

https://www.biospace.com/article/releases/bridgebio-pharma-announces-opportunity-for-accelerated-approval-pathway-in-limb-girdle-muscular-dystrophy-type-2i-lgmd2i-r9-based-on-glycosylated-alpha-dystroglycan-dg-levels-and-announces-first-patient-dosed-in-fortify-phase-3-study/