Chiesi’s flagship Fabry drug heads for FDA verdict in early ...

The FDA has started its review of Israeli biotech Protalix BioTherapeutics and partner Chiesi’s Fabry disease therapy pegunigalsidase alfa, setting up a possible approval by 27

Mallinckrodt’s liver disease drug terlipressin edges FDA adv...

An FDA advisory committee has narrowly voted in favour of approval of Mallinckrodt’s terlipressin for hepatorenal syndrome type 1 (HRS-1), after the agency questioned the findi

NICE backs NHS use of Takeda’s Adcetris for rare blood cance...

Takeda’s antibody-drug conjugate Adcetris can now be routinely available on the NHS to patients with a rare form of non-Hodgkin lymphoma (NHL), says NICE.

Sanofi hands back gene therapies to Oxford Biomedica

Oxford Biomedica’s long-running alliance with Sanofi over a pair of gene therapies for ophthalmic diseases has come to an end, after an internal review by the French drugmaker.

CSL, BrightInsight team up on digital health for rare diseas...

CSL Behring has signed a deal with US firm BrightInsight to develop digital health products to help patients with rare diseases manage their conditions.

Menarini bags blood cancer drug with $677M Stemline acquisit...