FDA Grants Fast Track Status to Soleno’s Investigational Therapy DCCR for PWS

FDA Grants Fast Track Status to Soleno’s Investigational Therapy DCCR for PWS

Soleno Therapeutics recently received fast track status from the U.S. Food and Drug Administration for diazoxide choline controlled-release (DCCR), its investigational treatment for Prader-Willi syndrome (PWS).

DCCR is an extended-release, salt formulation of diazoxide, taken as a once-daily tablet. Diazoxide is a potassium channel activator, capable of crossing the blood-brain barrier. It has been used as a first-line treatment in a variety of rare diseases, but has not been approved for PWS.

According to Soleno, DCCR has the ability to reduce hyperphagia, or excessive eating, as well as other hallmark symptoms in PWS patients.

Fast track designation is meant to accelerate the development and review of new medicines to provide quicker access to them for patients with life-threatening disorders who have unmet medical needs.

The new status will provide the company with additional support from the FDA on DCCR’s development plan, including appropriate data collection, design of clinical trials, and use of biomarkers to track progression of the disease.

“The receipt of Fast Track designation represents a significant milestone for Soleno and our DCCR clinical development program,” Anish Bhatnagar, MD, CEO of Soleno Therapeutics, said in a press release. “PWS is a devastating condition with high unmet medical need and, based on the data generated to date, we believe DCCR has the potential to address this critical treatment gap. Enrollment in our Phase 3 clinical trial for DCCR in PWS is ongoing at multiple sites in the U.S. With Fast Track designation, we look forward to continued collaboration with the FDA, with the goal of delivering DCCR to patients in need as expeditiously as possible.”

DCCR may also be eligible for the FDA’s accelerated approval and priority review, both of which would speed up its potential arrival on the market, as well as rolling review, if certain criteria are met. Rolling review would allow Soleno to submit sections of its new drug application as they are finished instead of waiting to submit the entire completed document prior to marketing approval.

In April, Soleno announced its plans for the clinical developmental of DCCR. It recently launched a randomized, placebo-controlled Phase 3 clinical trial (NCT03440814) to investigate the effectiveness of DCCR in children and adults with PWS, based on positive data from a Phase 2 study (PC025), which showed that the treatment led to a statistically significant reduction in hyperphagia.

The Phase 3 trial is evaluating DCCR’s effects on hyperphagia and body fat mass, as well as patients’ and caregivers’ perceptions. The trial is currently ran estimated 100 participants at several locations in the U.S.

Diazoxide choline previously received orphan drug designation for the treatment of PWS in both the U.S. and European Union.