FDA Approves Stealth BioTherapeutics’ Forzinity as First Treatment for Barth Syndrome After Rejection and Delay

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; rare disease; mitochondrial dysfunction; accelerated approval

FDA Grants Accelerated Approval to Stealth BioTherapeutics’ Forzinity, the First Treatment for Barth Syndrome

FDA approval; Stealth BioTherapeutics; Forzinity; elamipretide; Barth syndrome; mitochondrial disease; accelerated approval; rare disease; pediatric disease; clinical trial

Merck Wins FDA Approval for Subcutaneous Keytruda as Exclusivity Challenges Approach

Merck; Keytruda; subcutaneous injection; FDA approval; Keytruda Qlex; patent expiration; biosimilars; checkpoint inhibitors; solid tumors; cancer immunotherapy

VectorY Strikes $1.2B Option and License Pact with Shape Therapeutics for Brain-Penetrant Gene Therapy Delivery

VectorY Therapeutics; Shape Therapeutics; SHP-DB1; adeno-associated virus (AAV) capsid; brain-penetrant delivery; gene therapy; neurodegenerative diseases; Huntington’s disease; Alzheimer’s disease; biobucks; pipeline; milestone payments; vectorized antibodies

Lilly’s Mounjaro Shows Strong Results in Youth Type 2 Diabetes, Paving Way for Expansion

Mounjaro; tirzepatide; type 2 diabetes; children; adolescents; SURPASS-PEDS; A1C reduction; BMI reduction; clinical trial; Eli Lilly

Roche Acquires 89bio with $2.4 Billion Buyout to Enter FGF21 MASH Therapeutics Space

Roche; 89bio; acquisition; FGF21; pegozafermin; MASH; metabolic dysfunction-associated steatohepatitis; liver disease; biotech; contingent value rights; cardiometabolic diseases