The European Medicines Agency rejected Santhera’s Raxone as a treatment for Duchenne muscular dystrophy but the prospect of definitive data supporting the new indication means idebenone will still be available to patients in the UK under the early access scheme for promising new medicines.
The UK drug regulator has decided not to drop Santhera Pharmaceuticals’s drug Raxone (idebenone) from its early access to medicines scheme (EAMS) as a treatment for Duchenne muscular dystrophy, despite the European Medicines Agency saying the product should not be approved.
The EAMS designation for Raxone was granted last June and allows Santhera to make the drug available to patients in...
Eisai and Lilly explain why they disagree with today’s conclusion by the health technology assessment institute, NICE, that the benefits of their respective drugs for Alzheimer’s disease are too small to justify the additional cost to the National Health Service.
Highlights from Day Three of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for the FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.
There is a place for using external reference pricing to set prices of advanced therapies, but any such mechanism must take into account the specificities of the Brazilian market and health system.
Country recommends eights new drugs for approval, including six for rare diseases, and also announces macroeconomic policy the pharma industry says would enable some reimbursement prices to be raised.
Kumar takes over as acting director of the Center for Biologics Evaluation and Research’s Office of Therapeutic Products days after director Nicole Verdun was ousted.
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
While treatments for scleroderma are in development, none are approved for the condition that is associated with high morbidity and mortality. The European Medicines Agency says guidance on how to design development programs for the disorder is needed.
