Rare diseases

News

The FDA has added a warning label to bluebird bio's Skysona, after launching a probe into cases of blood cancer linked to the gene therapy.

News

Jazz Pharmaceuticals has become the first drugmaker to get FDA approval for a treatment for diffuse midline glioma with H3 K27M mutations.

News

Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.

News

Sanofi has paid $130 million upfront for Chinese rights to an Arrowhead Pharma drug for rare disease familial chylomicronaemia syndrome.

R&D

The second quarter of 2025 has seen a surge of clinical trial activity across the pharmaceutical and biotech landscape.

News

Merck gets another quick return on its $3.9bn SpringWorks takeover with EU approval of Ezmekly for tumours caused by neurofibromatosis type 1.