Rare diseases

News

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

R&D

Developing treatments for rare diseases poses many unique challenges that cannot be addressed by traditional study designs.

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.

News

AstraZeneca's light chain (AL) amyloidosis candidate anselamimab has failed a phase 3 readout, but showed efficacy in a patient subgroup.

News

LEO Pharma has licensed a medicine for a rare form of psoriasis from Boehringer Ingelheim, plugging a gap in its near-term product portfolio.