FDA

Sarepta Therapeutics is voluntarily pausing ELEVIDYS shipments in the U.S. to facilitate information exchange with the FDA and finalize safety labeling. This gene therapy treats Duchenne muscular dystrophy by delivering a micro-dystrophin gene. The pause will take effect July 22, 2025. The company emphasizes patient safety and maintaining a productive relationship with the FDA. ELEVIDYS is approved for Duchenne patients aged 4+ with confirmed DMD gene mutations.

Achieve Life Sciences priced a public offering of common stock and warrants at $3.00 per share to fund its cytisinicline program, targeting nicotine dependence. The offering, expected to close around June 30, 2025, aims to raise approximately $45 million, which will be used to advance cytisinicline through the FDA approval process. Cytisinicline, with Breakthrough Therapy designation, is designed to interact with nicotine receptors, potentially aiding smoking cessation.