Rare diseases

News

Pharvaris has reported positive data from a phase 3 trial of its drug for preventing HAE attacks, setting up marketing applications next year.

News

Merck's drug for tumours associated with rare disease neurofibromatosis type 1, Ezmekly, has been cleared for NHS use in children as young as two.

News

Ionis' Zanvastro has been approved by the FDA for Alexander disease, becoming the first disease-modifying therapy for the devastating rare disease.

News

Ultragenyx's therapy for the rare genetic disorder Angelman syndrome, apazunersen, has flunked a late-stage trial, crashing its share price.

News

Jazz Pharma has dipped its toe into the M&A water again, agreeing to buy rare epilepsy drug developer Actio Bio for $820m upfront.

News

Sobi chief executive Guido Oelkers is preparing his exit, but is still cutting deals, licensing a lymphoma drug from Innate for up to $580m.