Rare diseases

News

Spruce has bounced back from the failure of its lead drug last year, thanks to a licensing deal with BioMarin for a late-stage Sanfilippo drug.

News

Netherlands-based pharma group Norgine has expanded its rare disease portfolio with the acquisition of France's Theravia for an undisclosed sum.

News

The first biosimilars of AstraZeneca/Alexion's blockbuster complement C5 inhibitor Soliris have been launched in the US at a discount to the brand.

News

Amgen has bagged a second indication for its B cell-depleting therapy Uplizna after the FDA cleared it for rare inflammatory disease IgG4-RD.

News

Soleno Therapeutics' Vykat XR has become the first FDA-approved treatment for excessive eating in rare disorder Prader-Willi syndrome.

News

Novartis' oral complement inhibitor Fabhalta has become the first approved treatment for rare kidney disease C3G as it chases a $3bn sales target.