Agency reviewers believed the line between patient input and external intimidation had been crossed; early data from the Sarepta muscular dystrophy drug's flawed development program stoked patient community expectations and made regulatory review difficult.
FDA, patient advocates and rare disease drug developers should take away several lessons from the development and regulatory review of Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy drug Exondys 51 (eteplirsen).
FDA learned the challenges of integrating the patient voice in the regulatory decision-making process without its reviewers and staff being...
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
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.
<p>Executive Summary<p> <p>The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add six new products, incl/ding Ixchiq, Valneva's chikungunya virus vaccine</p>
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.
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