Orphan drugs

European conditional approval given for rare liver disease drug

The European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended a new drug to treat a rare and potentially lethal liver disease for conditional marketing approval across the EU.  Obeticholic acid (Ocaliva; Intercept Pharma) has been re…

NICE to fast track most cost-effective treatments

The National Institute for Health and Care Excellence (NICE) and NHS England have proposed changes to NICE’s drug appraisal process so that some treatments become available more quickly to patients.…

Heart failure drug Entresto wins Innovative Product at 2016 UK Prix Galien awards

Novartis has scooped one of the top 2016 UK Prix Galien Awards, considered to be the highest accolades that can be awarded to a pharmaceutical product. The Innovative Product Award was given to its heart failure treatment, Entresto (sacubitril/valsartan), the firs…

Obeticholic acid gets US approval for rare liver disease

The US Food and Drug Administration has approved a new drug called Ocaliva (obeticholic acid) for patients with primary biliary cholangitis. …

EMA recommends conditional approval of daratumumab

The European Medicines Agency has recommended the multiple myeloma treatment daratumumab for conditional marketing authorisation.…

Existing drugs could fight lethal viral infections

Researchers say repurposed drugs, such as bupicavaine, haloperidol and fluoxetine, could treat bunyavirus infection…

Human coagulation factor X recommended for approval in Europe

The European Medicines Agency is recommending that human coagulation factor X (Coagadex) should be authorised to treat the rare bleeding disorder factor X deficiency.…

FDA approves selexipag for pulmonary arterial hypertension

The US drugs safety regulator has approved selexipag to treat adults with pulmonary arterial hypertension, a chronic, progressive lung disease that causes high pressure in the arteries that connect the heart to the lungs.…

FDA approves treatment for inherited bleeding disorder

Recombinant von Willebrand factor, a treatment for an inherited bleeding disorder, has been approved by the US Food and Drug Administration.…

NICE rejects £360,000 therapy for rare paediatric bone disorder

A treatment for a rare childhood bone disorder has been provisionally rejected for NHS funding by the UK’s National Institute for Health and Care Excellence (NICE), which ruled the drug does not represent value for money.…