Rare diseases
Reports of seven deaths in patients who had taken Neurocrine Biosciences’ Vykat and the official termination of Aardvark Therapeutics’ Phase 3 trial after cardiac complications marked a tough day for the Prader-Willi syndrome community.
Mereo BioPharma’s asset, dubbed alvelestat, is set to enter Phase 3 development for AATD-LD in 2027.
BridgeBio Pharma’s transthyretin amyloidosis medicine Attruby is off to a strong launch, while Alnylam lowered full-year sales guidance for competitor Amvuttra.
The mid-stage win of Silence Therapeutics’ siRNA drug in polycythemia vera validates the therapeutic approach of similar assets in development—but also sets a high standard for therapies from Takeda, Agios and Disc Medicine.
Vaderis is hoping that engasertib will become the first approved medicine for hereditary hemorrhagic telangiectasia, a rare condition that can cause spontaneous bleeding.
A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.
Jazz Pharmaceuticals is reaching into its wallet once again, this time buying Actio Biosciences and an experimental therapy for KCNT1+ epilepsy, a form of the disease with no FDA-approved treatments.
BioMarin’s discontinued drug showed biomarker benefits in a Phase 3 study for ENPP1 deficiency but these failed to translate to clinical improvements.
After launching in April, Hunter syndrome drug Avlayah surprised analysts with a strong first quarter, as patients rush to get access to Denali Therapeutics’ new option.
In November 2025, one patient died after receiving Intellia Therapeutics’ investigational gene silencer for transthyretin amyloidosis.
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