US FDA's Stein Outlines Functions Of Rare Disease, Inborn Error Groups In New OND Division
Office of New Drugs director Peter Stein shed some light on how the newly established Division of Rare Diseases and Medical Genetics will operate following the completion of the OND reorganization, drawing mixed reactions from industry speakers.
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Rare Pediatric Disease Designations May Be Unavailable After July As Program Renewal Remains Uncertain
Without new legislation, US FDA said that designations, which are required to receive priority review vouchers, cannot be assured if requested after July. While the window for designations formally closes at the end of September when the authorization of the program expires, the agency is telling sponsors that it needs 60 days before that to review any requests. If a product does get a designation, it would need to secure approval by 30 September 2022 to gain a priority review voucher.
The FDA has confirmed the picks to head most of the new offices in the restructured drug review organization, with one notable exception. The choice for the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine is still pending, while one candidate – Hylton Joffe – will be serving as acting OND deputy director.
After US FDA's Oncologic Drugs Advisory Committee voted to delay approval of Karyopharm's multiple myeloma drug Xpovio, the company submitted some data from the Phase III BOSTON trial, which prompted the FDA to award an accelerated approval for a narrower indication.