Launching much faster than it told a US FDA advisory committee that it would, Sarepta is focusing on Duchenne muscular dystrophy patients about to turn six in the initial roll-out of its gene therapy Elevidys amid fears the wait for payer authorization could mean losing access to the treatment.
Suddenly, birthdays have taken on much more importance among Duchenne muscular dystrophy patients. The difference between age five and age six could mean the difference between receiving the latest gene therapy and not ... at least for now.
Sarepta Therapeutics, Inc.’s recently launched gene therapy Elevidys (delandistrogene moxeparvovec-rokl) is indicated only for ambulatory four- and five-year-olds with...
Cardiovascular and Renal Drugs Advisory Committee will consider whether open-label extension data from a randomized trial that failed its primary endpoint, along with a historical control comparison, are enough to support approval in the ultra-rare disease.
The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.
Under current law, drugs or biologics must receive rare pediatric disease designation before 1 October to be eligible for a priority review voucher. The sunset provisions have led to a spike in designation requests at the FDA and a push by pediatric and rare disease advocates for an extension, either through a continuing resolution or reauthorizing legislation.
A real-world evidence study also does not meet the regulatory standard for an adequate and well-controlled clinical investigation, the agency said. The FDA should allow the accelerated approval drug to remain available like other treatments that failed their confirmatory trials, Intercept said.
Revised recommendation is based on most recent data on circulating strains in US, where KP.3 is now dominant. Novavax sticks with plans targeting the JN.1 ‘parent strain.’
Pink Sheet reporter and editors discuss the US FDA’s changes to the COVID-19 vaccine indication and its revocation of the vaccine emergency use authorizations, as well as new vaccine coverage and reimbursement questions.
To make your document look professionally produced, Word provides header, footer, cover page, and text box designs that complement each other. For example, you can add a matching cover page, header, and sidebar. Click Insert and then choose the elements you want from the different galleries.
