ChemoCentryx has claimed FDA approval for its avacopan drug for a rare autoimmune disease, becoming the first orally-active drug that targets the complement C5 receptor.
People living with sickle cell disease (SCD) in England and Wales will be able to get access to treatment with Novartis' Adakveo, thanks to a special arrangement between the drugmaker and N
AstraZeneca's newly acquired Alexion will buy the remaining equity in rare disease specialist Caelum Biosciences in a deal that could be worth up to $500 million.
Biohaven's key pipeline drug verdiperstat has failed to have any Therapeutic effect in patients with the rare neurological disorder multiple system atrophy (MSA) – its lead indication – but
BioCryst Pharma's Orladeyo has been recommended by NICE as a treatment for the rare disease hereditary angioedema (HAE) in England, Wales and Northern Ireland.
There has been a fourth patient fatality in Astellas' clinical trial of its AT132 gene therapy for the rare disease X-linked myotubular myopathy (XLMTM), which has been halted twice due to