FDA Clears uniQure to File AMT-130 for Huntington’s Disease
NeuroMarket Pulse — June 17, 2026
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The FDA cleared uniQure to file AMT-130 for accelerated approval in Huntington’s disease — reversing its earlier demand for an entirely new pivotal trial — as Vedana Therapeutics launched with $46 million to chase the anti-PACAP migraine target and the IRA’s Part D redesign rewrote Medicare’s MS formularies.
Today’s top developments:
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What to Watch
- uniQure AMT-130 BLA (guided to Q3 2026) — With FDA alignment confirmed on an accelerated approval pathway, attention turns to uniQure’s Q3 2026 BLA submission. AMT-130’s RMAT, Breakthrough, and Fast Track designations support a likely priority review (~6 months), which could yield a 2027 advisory committee and approval decision. The required confirmatory trial — expected to use a concurrent standard-of-care control arm — will be a key commercial and valuation variable.
- BTK inhibitor race in MS — Roche’s fenebrutinib, with positive Phase 3 data in relapsing MS (FENhance) and primary progressive MS (FENtrepid), is expected to file in H1 2026; Sanofi’s tolebrutinib remains in FDA discussions after a December 2025 Complete Response Letter for non-relapsing SPMS; Novartis’s remibrutinib is still in Phase 3. Whichever files first enters a Medicare market reshaped by near-universal generic MS coverage.
- NICE reconsideration of lecanemab and donanemab — NICE’s appeal-ordered reconsideration of both anti-amyloid therapies is pending. Eisai’s ~$65M (£48M) UK manufacturing and cold-chain investment at its Hatfield site positions it for launch readiness against Lilly’s donanemab if the cost-effectiveness decision turns favorable.
- PACAP migraine market — Lundbeck’s bocunebart leads the anti-PACAP class with positive Phase 2 (PROCEED) data in treatment-refractory patients, while Vedana’s $46M runway buys Phase 1 entry at best. Watch whether PACAP validates as the “next CGRP” and whether NICE’s atogepant (AQUIPTA) acute-migraine recommendation accelerates the gepant class’s expansion beyond prevention.
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