Another sign that it could be approved, the FDA helps:
SOLENO THERAPEUTICS RECEIVES BREAKTHROUGH THERAPY DESIGNATION FROM U.S. FDA FOR DCCR (DIAZOXIDE CHOLINE) EXTENDED-RELEASE TABLETS IN PRADER-WILLI SYNDROME (PWS)
First Ever Breakthrough Designation for a Drug Being Developed for PWS
Designation is Based on Data from the Phase 3 Program for DCCR
Planned Submission of a New Drug Application (NDA) for DCCR Remains on Track for Mid-2024
REDWOOD CITY, Calif., April 29, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (Soleno) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia. The designation reflects the Agency’s determination that, based on an assessment of the preliminary data from the Phase 3 clinical development program, diazoxide choline may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.
“The granting of Breakthrough Therapy Designation, the first for a drug being developed for the treatment of PWS, marks another important milestone for our DCCR clinical development program,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “This important designation is confirmation that the FDA views PWS as a serious condition and is an indication of DCCR’s potential to be a safe and effective treatment for PWS. We remain focused on preparing our NDA submission for DCCR in PWS, which we continue to expect will occur in mid-2024.”
The FDA's Breakthrough Therapy Designation is intended to expedite the development and review of drugs in the U.S. that are intended to treat a serious condition, when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). With Breakthrough Therapy Designation, FDA provides intensive guidance and organizational commitment involving senior managers in a proactive, collaborative, cross-disciplinary review, and may also allow for priority review and other actions to expedite review.
Diazoxide choline has Orphan Drug Designation for PWS in the U.S. and E.U., as well as Fast Track Designation in the U.S.
https://investors.soleno.life/news-...-receives-breakthrough-therapy-designation-us
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