First in class narcolepsy medicine recommended for approval in Europe The first drug of its kind to treat patients with the rare but long-term sleep disorder narcolepsy is being recommended for use across the European Union.…
FDA approves Factor X therapy for rare bleeding condition The US Food and Drug Administration has approved a purified clotting factor for patients with hereditary Factor X deficiency.…
FDA approves drug for genetic condition that affects 20 patients worldwide The US Food and Drug Administration has approved uridine triacetate (Xuriden) for the treatment of orotic aciduria – a rare genetic hereditary metabolic disorder that affects just 20 patients worldwide. …
Asfotase alfa gets EU greenlight for rare bone condition The first treatment in Europe available for patients with the rare bone disease hypophosphatasia is a step closer following a recommendation from the medicines safety watchdog.…
First treatment for blind people with rare sleep disorder gets the go-ahead in Europe A drug to treat a rare sleep disorder almost exclusively affecting blind people has been recommended for authorisation by the European Medicines Agency.…
EMA recommends treatment for rare kidney condition The European Medicines Agency has recommended the approval of tolvaptan for the treatment of a rare inherited disease, autosomal dominant polycystic kidney disease.…
US FDA gives green light for thyroid cancer drug lenvatinib Lenvatinib will be available in the United States by the end of February 2015 after its approval was fast-tracked by the FDA.…