BridgeBio Achieves Enrollment Milestone for BBP-418 Study
BridgeBio Completes Enrollment of FORTIFY Study for BBP-418
- Study enrollment exceeded expectations, with topline data readout from the interim analysis anticipated in the next few years.
- BridgeBio sees a potential pathway for Accelerated Approval in the U.S. for BBP-418, utilizing a biomarker surrogate endpoint of glycosylated alpha-dystroglycan (?DG) during the interim analysis.
- If trials are successful, BBP-418 could become the first treatment approved for individuals with LGMD2I/R9 in the U.S.
PALO ALTO, Calif. — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a company focused on genetic diseases, declared the successful completion of enrollment for the FORTIFY clinical trial involving BBP-418 in individuals diagnosed with LGMD2I/R9.
FORTIFY is a comprehensive Phase 3 study that is randomized, double-blind, and placebo-controlled, specifically designed to evaluate the safety and effectiveness of BBP-418, an oral therapy aimed at treating LGMD2I/R9. An interim analysis is scheduled for 12 months, focusing on glycosylated ?DG as a surrogate endpoint that could support the potential for Accelerated Approval. In 36 months, the primary endpoint will be assessed using the North Star Assessment (NSAD), a crucial measure for limb-girdle muscular dystrophies, to validate the efficacy of BBP-418.
CEO Kelly Brazzo from the CureLGMD2i Foundation highlighted the impact of LGMD2I/R9 on patients' daily lives, stating that those dealing with this progressive condition often find themselves losing independence in performing daily activities. The swift enrollment in this trial represents a beacon of hope for affected individuals and their families.
Dr. Douglas Sproule, Chief Medical Officer of ML Bio Solutions, emphasized the significance of this rapid enrollment, saying it affirms the urgent need for effective treatments for patients facing LGMD2I/R9. Thanks to productive discussions with the FDA, they're optimistic about the possibility of pursuing Accelerated Approval based on the biomarker assessment during the interim analysis. If successful, BBP-418 could mark a significant milestone as the first approved disease-modifying therapy for this condition in the U.S.
The BBP-418 drug has already received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA, in addition to Orphan Drug Designation from the European Medicines Agency (EMA). Approval for BBP-418 may also allow BridgeBio to qualify for a Priority Review Voucher.
Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
LGMD2I/R9 is classified as a monogenic autosomal recessive disease, arising from mutations that lead to a partial loss of function of the fukutin-related protein (FKRP) gene. These mutations interfere with the proper glycosylation of ?DG, a key protein stabilizing muscle cells. The disease usually manifests as skeletal myopathy, which often begins in the lower limbs and can progress to involve upper limbs, respiratory muscles, and cardiac muscles later on.
Individuals with a homozygous L276I genotype typically begin to show symptoms in late childhood, facing significant mobility challenges, such as loss of independent walking (25%) and requiring assisted ventilation (10%) in adulthood, along with a 30% rate of cardiomyopathy. Those with other FKRP mutations usually have onset earlier in childhood, leading to a more aggressive disease progression and a greater risk of cardiac complications.
About BridgeBio Pharma, Inc.
BridgeBio seeks to discover and deliver transformative therapies for patients afflicted by genetic disorders. Founded in 2015, the company is committed to creating solutions that capitalize on developments in genetic medicine, ensuring that patients receive innovative treatments as swiftly as possible. The team comprises experienced professionals dedicated to the research and development of genetic disease therapeutics.
What to Expect Next
As the FORTIFY study progresses, further data will be collected and shared with the medical community. The focus will be on ensuring the safety and efficacy of BBP-418 while remaining transparent about the trial outcomes.
Community Engagement
BridgeBio values the active participation of patients, families, and investigative sites in clinical trials. Their involvement is crucial for the advancement of new therapies aimed at addressing unmet medical needs.
Frequently Asked Questions
What is the FORTIFY study?
The FORTIFY study is a Phase 3 clinical trial investigating the safety and efficacy of the investigational drug BBP-418 for treating LGMD2I/R9.
What is BBP-418?
BBP-418 is an oral therapy developed by BridgeBio Pharma intended to treat patients with LGMD2I/R9.
What are potential outcomes from the FORTIFY study?
A successful trial could lead to the Accelerated Approval of BBP-418, providing much-needed treatment options for individuals with LGMD2I/R9.
How does LGMD2I/R9 affect patients?
LGMD2I/R9 is a progressive muscle-wasting condition that impacts day-to-day activities and may lead to severe mobility and health challenges.
How can patients and families get involved?
Patients and families can participate in clinical trials and stay informed about ongoing research by engaging with organizations like BridgeBio and relevant foundations.
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