Rare Diseases
From a biofoundry network and pharma industry backed AI research organization to a small-molecule repurposed drug in Duchenne’s muscular dystrophy, experts discuss a number of advances underway as India seeks to move up the innovation ladder.
The company plans to take Aqneursa to regulators in the US, Europe and elsewhere for ataxia-telangiectasia (A-T), which currently lacks any approved therapy.
HUTCHMED sees good potential for its Syk inhibitor in autoimmune setting following positive topline Phase III results, with China NDA planned in first half.
The Niemann-Pick disease type C drug is off to a good start in the US but Europe may be even more lucrative, CEO Neil McFarlane tells attendees at the J.P. Morgan meeting.
A deal for the Duchenne muscular dystrophy treatment with Nxera is valued at up to $205m.
Ersodetug missed its Phase III primary endpoint in congenital hyperinsulinism but thinks building on data showing pharmacologic activity may offer a path to approval.
CEO and co-founder Tim Van Hauwermeiren will transition to chairman of the board of directors.
In its second deal of 2025 building its enzyme replacement therapy portfolio, BioMarin says Amicus’s two marketed products may grow into blockbusters with greater geographic reach.
The US FDA approved Uplizna (inebilizumab) for generalized myasthenia gravis, an increasingly crowded market. Amgen believes it can compete due to the CD19-targeting antibody’s durable efficacy with twice-yearly dosing.
Novartis presented Phase III results for anti-BAFF/ADCC antibody ianalumab in the second-line treatment of immune thrombocytopenia but will wait for first-line data to file for approvals in 2027.
Dyne’s data from a larger Phase I/II trial cohort showed much greater dystrophin production with z-rostudirsen (DYNE-251) than seen with Sarepta’s Exondys 51 and some functional gains.
With three approved drugs and volixibat in pivotal studies for two cholestatic liver diseases, Mirum is betting $570m in cash and equity to pick up Bluejay’s Phase III hepatitis D candidate.
Pharvaris’s oral bradykinin B2 antagonist hit all endpoints in a Phase III study in hereditary angioedema attacks, meaning the drug could be competing soon with Kalvista’s Ekterly.
The pivotal Phase III trial met the primary and secondary endpoints, showing skeletal and cardiac benefits and supporting deramiocel as a treatment for Duchenne cardiomyopathy.
US FDA approval of the intrathecal formulation of Zolgensma means many more patients with spinal muscular atrophy will be eligible for the gene therapy.
The Phase III portion of the Phase II/III RISE UP study in sickle cell disease met the hemoglobin response primary endpoint, but not the sickle cell pain crisis reduction co-primary endpoint nor a secondary endpoint on reducing fatigue.
Five-year Agamree data shows the novel steroid is a safer and equally effective treatment to standard of care deflazacort or prednisone, the Swiss biotech argues.
After two deaths tied to the gene therapy, Sarepta and the FDA agreed to new labeling for Elevidys, limiting treatment to ambulatory Duchenne muscular dystrophy patients.
Zydus looks to fill up its coffers ahead of the next round of acquisitions, likely in the specialty drugs space, while US FDA approvals for generics to Lynparza and Vumerity and its first approvals in China and Canada point to a healthy pipeline
The German group is looking at ‘single digit billion’ opportunities, chief financial officer Helene von Roeder tells Scrip.