Recent regulatory decisions mark significant progress in oncology and rare disease treatments. The FDA approved Stealth BioTherapeutics’ elamipretide as the first therapy for Barth syndrome after initially rejecting the drug. Merck received FDA approval for Keytruda Qlex, a subcutaneous immunotherapy for solid tumors enabling a drastically reduced administration time. Additionally, Ionis Pharmaceuticals announced positive pivotal trial results for its antisense oligonucleotide in Alexander disease, planning to seek FDA approval soon. These approvals and trial advances underline ongoing momentum in precision and rare disease therapeutics.