Alexion’s Soliris follow-up Ultomiris snares EU approval

Pfizer acquires Therachon and its dwarfism drug

Pfizer has announced it will pay $340 million upfront to snap up rare disease biotech Therachon, which is developing treatments for a form

Fulcrum repurposes failed GSK drug for rare disease

GSK’s failed cardiovascular drug losmapimod looks like it might get a new lease of life after being picked up by biotech Fulcrum Therapeutics, who want to repurpose the drug to treat the ra

Second death in gene therapy trial prompts investigation

A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte

Ipsen launches new syringe for rare disease blockbuster

Ipsen will be launching a new pre-filled syringe for its blockbuster rare disease drug Somatuline (lanreotide) in the EU.

Sanders calls on FDA to allow low-cost rivals to $375k Catal...

US Senator Bernie Sanders says the FDA should allow pharmacies and generic manufacturers to make low-cost copies of Catalyst Pharma’s rare disease drug Firdapse, which is at the heart of a