==BridgeBio Pharma (BBIO) : Breakthrough Therapy Designation to oral infigratinib

 

BridgeBio Pharma announces that the FDA has granted Breakthrough Therapy Designation to oral infigratinib under development for children with achondroplasia

• Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA's requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s).
• BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and if approved, infigratinib could be the first-in-class oral therapeutic option for children living with achondroplasia.
• In Cohort 5 of PROPEL 2 (0.25 mg/kg/day), oral treatment with infigratinib resulted in a statistically significant and sustained increase in annualized height velocity (AHV), with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015) and statistically significant improvement in body proportionality at Month 18.

====BridgeBio Pharma (BBIO) : positive results from Phase 3 ATTRibute-CM study

 

BridgeBio announces consistently positive results from Phase 3 ATTRibute-CM study of acoramidis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM)

PALO ALTO, Calif., July 17, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), is a commercial-stage biopharmaceutical company focused on genetic diseases and cancers. Today, alongside the dedicated physicians and courageous patients who participated, the Company reports positive results from ATTRibute-CM, its Phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). BridgeBio will host an investor call on July 17, 2023 at 8:00 am ET to discuss these results.