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 accepted Roche's approval application for fenebrutinib, an experimental multiple sclerosis pill, under a priority review with a six-month timeline, covering both relapsed and primary progressive forms of the disease. The application rests on three late-stage studies that met their main objectives, but approval chances could be complicated by an imbalance in deaths, eight among fenebrutinib recipients versus one on Aubagio in two Phase 3 trials, and a case of liver enzyme elevations meeting Hy's law. Roche did not say when a decision might come.
Why it matters: Fenebrutinib could become the first BTK inhibitor approved for two major forms of MS despite unresolved safety concerns.
The MHRA will suspend the use, supply and sale of Avacopan Vifor (previously Tavneos) to new patients from 1 September 2026, after concluding that data integrity problems in the pivotal trial mean the study can no longer demonstrate efficacy. The drug, authorised on 6 May 2022 with rituximab or cyclophosphamide for severe, active GPA or MPA in adults, will be supplied only to existing patients during a six-month managed withdrawal, with the UK marketing authorisation to be revoked on 1 March 2027. Clinicians should not start new patients on the medicine and should review current patients for alternative treatments.
Why it matters: UK regulator suspends and plans to revoke a vasculitis drug's authorisation over unreliable pivotal trial data.
The FDA has approved brepocitinib, brand name Lisraya, from Roivant and its subsidiary Priovant, for dermatomyositis, a rare autoimmune disease marked by muscle weakness and a painful skin rash. The once-daily pill affects a U.S. patient population of 40,000 to 70,000 and offers an alternative to high-dose steroids and other immune-suppressing drugs.
Why it matters: FDA approval of a new oral therapy for a rare autoimmune disease with no tailored standard of care.
The FDA has approved Priovant Therapeutics' brepocitinib, to be marketed as Lisraya, a once-daily pill for adults with dermatomyositis, making it the first targeted therapy approved in the disease. The approval rests on the phase 3 Valor trial of 241 patients, where the high dose achieved a mean Total Improvement Score of 46.5 versus 31.2 for placebo, with 55% of treated patients reaching moderate or better improvement with minimal or no steroid use versus 30% on placebo. The Roivant-backed company, formed in 2022 with a Pfizer deal, is also testing the dual TYK2/JAK1 inhibitor in phase 3 noninfectious uveitis and cutaneous sarcoidosis.
Why it matters: First FDA approval of a targeted therapy for dermatomyositis, for a company's debut drug with further late-stage indications underway.
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.
The European Commission has revoked the EU marketing authorization for Tavneos, used for active granulomatosis with polyangiitis and microscopic polyangiitis in ANCA-associated vasculitis, adopting a June CHMP recommendation. Regulatory documents detail 'serious breaches' of good clinical practice in the pivotal phase 3 Advocate study, where sponsor personnel allegedly saw unblinded efficacy data after database lock and re-adjudicated nine patients, turning a non-significant result into statistical significance at week 52. CHMP also cited fatal cases of drug-induced liver injury and vanishing bile duct syndrome. CSL Vifor, the EU marketing operator, will implement the withdrawal, while Amgen, which bought developer ChemoCentryx for $3.7 billion in 2022, disagrees and continues fighting a separate FDA withdrawal request in the U.S.
Why it matters: EU regulators detail data-integrity breaches behind the Tavneos withdrawal while Amgen contests parallel U.S. action.