BridgeBio advances acoramidis data and files BBP-418 NDA in rare diseases
BridgeBio Pharma reported long term acoramidis data in transthyretin amyloid cardiomyopathy and submitted a US FDA NDA for BBP-418 in LGMD2I/R9. BBP-418 could become the first approved therapy for the disease if cleared.
BridgeBio Pharma reported long term data for acoramidis (Attruby) in transthyretin amyloid cardiomyopathy, showing sustained efficacy, safety and quality of life outcomes over several years. The company also submitted a New Drug Application to the US FDA for BBP-418, supported by Phase 3 data in limb-girdle muscular dystrophy type 2I/R9.
For ATTR-CM, the company highlighted the role of early and continuous treatment for patients, citing ongoing unmet medical needs with existing options. The long term acoramidis data give physicians and payers more visibility on durability of effect, safety and quality of life over more than four years, which can be important when treatment is chronic.
For LGMD2I/R9, the BBP-418 filing moves the program into a regulatory review phase. If cleared by regulators, BBP-418 would be the first approved therapy for LGMD2I/R9, addressing a patient group with no current approved treatments.
BBP-418 has orphan, Fast Track and Rare Pediatric Disease designations. The company said updates on European regulatory discussions, plans to study younger LGMD2I/R9 patients and additional indications such as LGMD2M/2U will also shape views on the long term market opportunity for BBP-418.
BridgeBio said these two updates push both its current product and late-stage pipeline forward at the same time, with rare cardiovascular and neuromuscular conditions at the center of its story.