Rare diseases

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.

News

Partner's Bizengri has been approved for a rare form of bile duct cancer, two days after being awarded a national priority review voucher.

News

Patients in the EU with the ultra-rare disease WHIM syndrome now have their first approved treatment, Norgine's Xolremdi.

News

Chiesi has boosted its rare disease business with an agreement to buy KalVista and Ekterly, its oral drug for treating attacks in rare disease HAE.

News

Intellia has reported the first-ever phase 3 results with an in vivo gene-editing therapy, CRISPR-based lonvo-z for hereditary angioedema.