Advanz Pharma is urging the European authorities to consider the “thousands” of primary biliary cholangitis (PBC) patients in the EU who will be left without treatment if the European Commission withdraws the conditional marketing authorization for its rare disease drug, Ocaliva (obeticholic acid), on the basis of a recommendation from the European Medicines Agency, according to Steffen Wagner, Advanz’s chief executive officer.
EU Ocaliva Withdrawal Recommendation Based On ‘Flawed’ Studies
Overreliance on “problematic” trials while overlooking the value of real-world evidence could affect the development of other rare disease treatments, according to Advanz’s CEO.
More from Europe
• By
Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.
• By
The weight management drug, Mysimba, continues to demonstrate a positive benefit-risk balance but the data available are not sufficient to fully determine the cardiovascular safety beyond 12 months. Meanwhile, Currax this month announced the publication of a peer-reviewed study of Mysimba that followed patients for over 4.7 years and found no evidence of excess cardiovascular risk.
• By
Health technology assessment body NICE said it has taken on feedback about the implications of allowing higher cost-effectiveness thresholds for some medicines after senior health economists offered diverging views on its methods.
• By
The European Medicines Agency has recommended five drugs for EU-wide approval , including Averoa’s Xoanacyl for concomitant hyperphosphatemia. Two companies have withdrawn their marketing authorization applications.
More from Geography
• By
Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.
• By
The Brazilian medicines regulator will also offer more clarity on the requirements for radiopharmaceuticals that are exempt from registration.
• By
South Korea announces planned revisions to a government scheme to designate "innovative" biopharma companies amid allegations of "discrimination" against foreign firms.