FDA Accepts NDA for Soleno's DCCR in Prader-Willi Syndrome
FDA Acceptance of NDA for DCCR in Prader-Willi Syndrome
Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for rare diseases, has recently announced that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for DCCR (Diazoxide Choline) extended-release tablets. This formulation aims to address the complex needs of patients diagnosed with Prader-Willi syndrome (PWS), particularly in individuals four years and older who experience hyperphagia—a life-threatening condition characterized by an insatiable appetite.
PDUFA Target Action Date
The FDA has granted Priority Review for this application, assigning a Prescription Drug User Fee Act (PDUFA) target action date later this year. This designation signifies that the FDA recognizes the potential for DCCR to improve treatment outcomes for patients suffering from PWS. Additionally, an advisory committee meeting is planned to further discuss the application, a standard procedure that allows for expert input on drug efficacy and safety.
The Importance of DCCR for PWS
“Prader-Willi syndrome is a severe, rare condition that significantly affects the quality of life for both patients and their families,” stated Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. The acceptance of the NDA marks a pivotal achievement, reflecting the FDA’s acknowledgment of the serious nature of PWS. Soleno appreciates the continued support from the PWS community, which includes patients, caregivers, and advocacy organizations dedicated to improving lives affected by this disorder.
PWS: A Brief Overview
According to the Prader-Willi Syndrome Association USA, PWS occurs in approximately one in every 15,000 live births. The syndrome is often marked by chronic hyperphagia that can result in serious health risks, including obesity, diabetes, and cardiovascular issues. The disorder also encompasses a range of additional symptoms that can severely impact an individual's well-being. These include behavioral challenges, cognitive impairments, and physical characteristics such as low muscle tone and short stature.
Survey Insights on Patient Needs
A global survey by the Foundation for Prader-Willi Research found that a staggering 96.5% of parents and caregivers rated hyperphagia as one of the most critical symptoms requiring management through new medication. Despite the prevalent challenges associated with PWS, there are currently no approved medicines available that specifically target the hyperphagia, metabolic issues, or behavioral concerns linked to the condition.
Understanding DCCR (Diazoxide Choline) Extended-Release Tablets
DCCR represents a novel, proprietary extended-release formulation that provides a once-daily dosage of diazoxide choline, a crystalline compound derived from diazoxide. The parent compound has a long history of use in treating rare diseases in various age groups but is not yet approved specifically for PWS. Soleno has rigorously developed and protected the therapeutic applications of diazoxide through extensive patent filings.
Clinical Development and Promising Results
The development program for DCCR is robust, founded on data gathered from five completed Phase 1 studies and three Phase 2 studies, including one focused on patients with PWS. The evidence generated during these studies points to DCCR’s potential not only in alleviating hyperphagia but also in mitigating other symptoms such as aggressive behaviors and unfavorable metabolic parameters. This underscores Soleno's commitment to advancing innovative treatments for PWS.
Soleno Therapeutics: Committed to Rare Diseases
As a leader in the development of therapeutics for rare conditions, Soleno Therapeutics aims to bring hope and relief to patients with unmet medical needs. The NDA for DCCR is currently under the FDA's review, and the designation of Priority Review indicates Soleno's intent to expedite the availability of effective treatments for patients and their families affected by PWS.
Corporate Contact Information
The company can be reached for further inquiries 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.
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