Rare diseases

News

Dren Bio has closed a sizeable private financing round, raising $350m for pivotal trials of a possible first-in-class therapy for rare blood cancers.

News

Egetis has launched the first-ever therapy for the ultra-rare disease MCT8 deficiency in the US, with a list price of $950,000 per year.

News

AstraZeneca has staked a claim to the increasingly crowded IgAN market with an accelerated approval from the FDA for Ultomiris, its fifth indication.

News

Merck KGaA is slated to acquire UK-based Silence Therapeutics in a $1.3bn deal that would give it a late-stage drug for polycythemia vera.

News

Shionogi has reached a $2bn agreement to buy Anglo-US biotech IntraBio and its recently approved Aqneursa therapy for two rare genetic disorders.

News

CSL has licensed rights to a potentially first-in-class anti-claudin-1 antibody developed by Alentis in a deal that could be worth up to $1.55bn.