Soleno Therapeutics has announced that the FDA will accept additional data from their DESTINY PWS and C602 extension study to determine if the data generated are sufficient to support a potential New Drug Application (NDA) for diazoxide choline controlled-release (DCCR) tablets for people with Prader-Willi syndrome.
This past March, the FDA communicated that an additional controlled clinical trial to support an NDA submission for DCCR in PWS. In response to this decision, a Type B meeting was conducted on June 11th with the Division of Diabetes, Lipids and Obesity. Representatives from FPWR and PWSA | USA attended the meeting to help provide the perspective of the patient community, as well as the family of a DCCR trial participant.
While the FDA continues to assert that based on the data they have seen to date, an additional clinical trial is necessary for the submission of an NDA, they have strongly encouraged Soleno to submit the available data and clinical study reports for the Company’s Phase 3 trial, DESTINY PWS (C601), and its long-term, open-label extension study (C602) to allow them to assess if these studies may provide adequate evidence of safety and efficacy to support the submission of an NDA.
“We are continuing our dialogue with the FDA and remain focused on our goal of achieving approval for DCCR for the treatment of PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “We appreciate the opportunity to include the patient voice in our discussions with the Division. We intend to submit additional data from DESTINY PWS and the C602 extension study to the Agency before the end of the third quarter. The FDA has agreed to review these data to determine if the totality of data generated to date are sufficient to support a potential NDA submission.”
The Phase-3 DESTINY PWS study, completed in June 2020, included 127 participants with PWS ages 4 and older. In the Phase 3 study, DCCR showed promise in addressing hyperphagia, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters.