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

After a no in sickle cell, NICE OKs Casgevy for thalassaemia

Vertex and CRISPR Therapeutics have won the backing of NICE for their gene-editing therapy Casgevy as a treatment for the blood disorder beta thalassaemia, a few months after turning it dow

Pfizer reaches deal with NICE on Vyndaqel for ATTR-CM

Three years after turning it down for being too expensive, NICE has backed the use of Pfizer’s Vyndaqel as a treatment for transthyretin amyloidosis with cardiomyopathy (A

Lifeline for Wolman disease kids as NICE backs Alexion drug

Infants with a rare and fatal congenital disorder called Wolman disease in England and Wales will soon have access to a new drug via the NHS that can dramatically extend t

One year on, ABPI asks why IMF hasn't funded any drugs

The Innovative Medicines Fund (IMF), set up in the UK last year to fast-track NHS access to promising new drugs, still hasn't provided any funding for a new medicine, and

Innovative Medicines Fund and the opportunity for ICSs to mo...