FDA's CDER lists recent rare disease drug approvals on its Accelerating Rare Disease Cures Program page. In 2026 alone these include Camzyos for an inherited heart condition in children, Emcitate for MCT8 deficiency, Isembyld for muscle loss in spinal muscular atrophy, and Zanvastro for Alexander Disease, among others. The list spans 2021 to 2026 and links to the underlying FDA news releases.
The FDA has approved Priovant Therapeutics' Lisraya (brepocitinib) tablets, the first oral treatment indicated for dermatomyositis in adults. The once-daily JAK/TYK2 inhibitor was supported by a phase 3 placebo-controlled study of 241 adults, in which the 30 mg dose produced a higher average Total Improvement Score at week 52 and allowed more patients to cut corticosteroid use. It carries a boxed warning for serious infections, mortality, malignancies, cardiovascular events and thrombosis, and was granted Orphan Drug and Priority Review designations.
Why it matters: First FDA-approved oral therapy for dermatomyositis, a rare disease with few treatment options.