Rare diseases

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.

News

Amgen is preparing to file a third indication for Uplizna, as a twice-yearly therapy for generalised myasthenia gravis, following new phase 3 data.

News

Spain has agreed to reimburse Orchard Therapeutics' ex vivo gene therapy for the rare childhood disease metachromatic leukodystrophy.