FDA Launches New Priority Review Voucher Program Aligned with National Priorities

FDA; priority review voucher; CNPV; biopharma; drug approval; national priorities; accelerated review; unmet public health needs

European Commission’s Reconsideration of Translarna’s Marketing Authorization: A Critical Turning Point for Duchenne Muscular Dystrophy Treatment

Translarna, Duchenne muscular dystrophy, European Commission, EMA, CHMP, conditional marketing authorization, drug approval, regulatory decisions

FDA Raises Concerns Over Stealth BioTherapeutics’ Ultra-Rare Disease Drug Ahead of Advisory Committee Meeting

FDA, Stealth BioTherapeutics, elamipretide, Barth syndrome, ultra-rare disease, advisory committee meeting, drug approval, efficacy concerns