Rare diseases

News

Advanz wins stay on EU revocation of Ocaliva license

There's been another twist to the tale of Advanz Pharma's efforts to keep its primary biliary cholangitis (PBC) therapy Ocaliva on the market in the EU.

News

Novartis' oral PNH drug Fabhalta rolls out in England, Wales

Novartis' targeted Factor B inhibitor Fabhalta – the first oral monotherapy for rare disease paroxysmal nocturnal haemoglobinuria (PNH) – has been cleared by NICE for use by the NHS in Engl

News

Advanz Pharma's bid to keep Ocaliva on EU market fails

The European Commission has followed the advice of its human medicines committee and revoked the conditional marketing authorisation for Advanz Pharma's Ocaliva therapy for primary biliary

News

Rare disease drug from LEO's Timber felled by failed study

Just a few months after buying Timber Pharma, LEO Pharma has revealed that the main drug it acquired as part of the deal has failed a phase 3 trial.

News

PBC drug choices rise in US with OK for Gilead's seladelpar

At the start of the year, there had been no new therapies for rare cholestatic liver disease primary biliary cholangitis (PBC) in the US for almost a decade.

News