Soleno Therapeutics (SLNO) FDA Announcement summary

Introduction and purpose

• FDA approved VYKAT XR (diazoxide choline extended-release) as the first treatment for hyperphagia in adults and children aged four and older with Prader-Willi syndrome (PWS).

• The approval addresses a significant unmet need for PWS patients and their families, who previously had no approved therapies for hyperphagia.

• PWS is a rare genetic disorder with a birth incidence of 1 in 15,000 and a global prevalence of 300,000–400,000 individuals.

Details of approval or decision

• VYKAT XR is indicated for hyperphagia in PWS patients aged four and above, with no box warnings, no diabetes contraindications, and no REMS requirement.

• The drug is available in three tablet strengths for weight-based dosing and is taken orally once daily.

• The label specifies VYKAT XR should not be substituted with diazoxide oral suspension due to different pharmacokinetics.

• Approval is based on a comprehensive clinical trial program, with no exclusions for severity of hyperphagia.

• Expected U.S. availability in April 2025.

Impact on industry and stakeholders

• The approval is seen as a historic milestone for the PWS community, offering hope and relief to patients and caregivers.

• The addressable U.S. market is estimated at 10,000 diagnosed individuals with PWS aged 4+ and hyperphagia.

• Advocacy groups and caregivers have shown high engagement and support for the launch.

• Commercial strategy targets high-volume prescribers, especially pediatric and adult endocrinologists and psychiatrists.

• Payer engagement underway to secure coverage, with a payer mix of commercial, Medicare, and Medicaid.