FDA Accepts NDA for DCCR in Prader-Willi Syndrome
Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a biopharmaceutical company focused on creating innovative treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for DCCR (Diazoxide Choline) extended-release tablets. This formulation is specifically designed to meet the complex needs of individuals diagnosed with Prader-Willi syndrome (PWS), especially those aged four and older who suffer from hyperphagia, a serious condition characterized by an uncontrollable appetite.
PDUFA Target Action Date
The FDA has granted Priority Review status to this application, setting a target action date under the Prescription Drug User Fee Act (PDUFA) for later this year. This designation indicates that the FDA recognizes DCCR's potential to significantly improve treatment outcomes for patients with PWS. Furthermore, an advisory committee meeting is scheduled to discuss the application in detail, a standard practice that allows experts to weigh in on the drug's efficacy and safety.
The Significance of DCCR for PWS
“Prader-Willi syndrome is a serious and rare condition that greatly impacts the quality of life for both patients and their families,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. The acceptance of the NDA is a significant milestone, highlighting the FDA's recognition of the serious challenges posed by PWS. Soleno is grateful for the ongoing support from the PWS community, which includes patients, caregivers, and advocacy groups dedicated to enhancing the lives of those affected by this disorder.
PWS: A Brief Overview
According to the Prader-Willi Syndrome Association USA, PWS affects approximately one in every 15,000 live births. The syndrome is often characterized by chronic hyperphagia, which can lead to severe health complications such as obesity, diabetes, and cardiovascular problems. In addition to these risks, PWS encompasses a variety of other symptoms that can profoundly affect an individual's overall well-being, including behavioral issues, cognitive challenges, and physical traits like low muscle tone and short stature.
Survey Insights on Patient Needs
A global survey conducted by the Foundation for Prader-Willi Research revealed that an overwhelming 96.5% of parents and caregivers consider hyperphagia to be one of the most critical symptoms that needs to be managed with new medication. Despite the significant challenges posed by PWS, there are currently no approved treatments that specifically address hyperphagia, metabolic issues, or behavioral concerns related to the syndrome.
Understanding DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR is a new, proprietary extended-release formulation that allows for a once-daily dose of diazoxide choline, a crystalline compound derived from diazoxide. While the parent compound has a long history of use in treating rare diseases across various age groups, it has not yet been approved specifically for PWS. Soleno has diligently developed and protected the therapeutic applications of diazoxide through extensive patent filings.
Clinical Development and Promising Results
The development program for DCCR is comprehensive, based on data from five completed Phase 1 studies and three Phase 2 studies, including one that specifically focused on patients with PWS. The findings from these studies suggest that DCCR has the potential not only to alleviate hyperphagia but also to reduce other symptoms such as aggressive behaviors and adverse metabolic parameters. This reinforces Soleno's commitment to advancing innovative treatments for PWS.
Soleno Therapeutics: Committed to Rare Diseases
As a frontrunner in developing therapeutics for rare conditions, Soleno Therapeutics is dedicated to providing hope and relief to patients with unmet medical needs. The NDA for DCCR is currently under review by the FDA, and the Priority Review designation reflects Soleno's goal of accelerating the availability of effective treatments for patients and families affected by PWS.
Corporate Contact Information
For further inquiries, the company can be contacted through:
Brian Ritchie
LifeSci Advisors, LLC
212-915-2578
Frequently Asked Questions
What is DCCR?
DCCR, or Diazoxide Choline, is an extended-release tablet designed to treat hyperphagia associated with Prader-Willi syndrome.
What does FDA Priority Review mean?
Priority Review is granted to applications that have the potential to significantly enhance patient treatment, allowing for an expedited review process.
What is the target action date for the DCCR NDA?
The target action date for the DCCR NDA under the Prescription Drug User Fee Act is set for December 27, 2024.
How common is Prader-Willi Syndrome?
Prader-Willi Syndrome occurs in approximately one in every 15,000 live births, making it a rare genetic disorder.
What are the main symptoms of PWS?
The most notable symptom of Prader-Willi syndrome is hyperphagia, accompanied by other symptoms like cognitive challenges, low muscle tone, and behavioral issues.
