Rare diseases

News

Sanofi's neurology pipeline has had a hole blown in it after a phase 3 trial of riliprubart in rare disease CIDP was stopped for futility.

News

The FDA has laid out a framework to use 'prior knowledge' on manufacturing, non-clinical, and clinical data to support cell and gene therapy filings.

News

The SARCO41 study suggests Lilly's Verzenio could become the first targeted therapy for rare, aggressive tumour dedifferentiated liposarcoma.

News

Trials of AstraZeneca's anselamimab and Regeneron's Lynozyfic could deliver much-needed new therapeutic options for rare disease AL amyloidosis.

News

After a phase 2 readout, Sanofi is charting a route to market for AATD therapy efdoralprin alfa, acquired as part of its $2.2bn takeover of Inhibrx.

News

AstraZeneca may struggle to differentiate eneboparatide, a hypoparathyroidism drug from its $800m takeover of Amolyt, from Ascendis' rival Yorvipath.