CLDF welcomes today’s news that Odebixivat (brand name Bylvay) meets the Scottish Medicines Consortium definition of an ultra-orphan medicine, ie a medicine to treat an extremely rare condition.
The drug is already licensed for use in England for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients who are six months or older. Today’s announcement means that if your healthcare professional thinks that odevixibat is the right medicine for your child, they should be able to have the treatment on the NHS in Scotland within the ultra-orphan pathway.
More information about Odebixivat and the ultra-orphan pathway is available here: AE odevixibat (Bylvay) SMC2411
