Soleno Therapeutics Makes History with FDA Approval of VYKAT XR for Prader-Willi Syndrome

In a groundbreaking move that could redefine the landscape of rare disease treatments, Soleno Therapeutics has secured U.S. FDA approval for VYKAT XR (diazoxide choline) extended-release tablets. This approval marks the first-ever therapy specifically targeting hyperphagia in individuals with Prader-Willi syndrome (PWS), a rare genetic disorder characterized by insatiable hunger and life-threatening obesity. The significance of this milestone cannot be overstated, as it addresses a critical unmet need in the PWS community, where previously there were no approved treatments for this debilitating symptom.

The approval of VYKAT XR is based on robust clinical evidence from a Phase 3 randomized withdrawal study, which demonstrated statistically significant efficacy. Patients who switched to a placebo showed a worsening of hyperphagia compared to those who continued on VYKAT XR. This strong clinical data, coupled with a well-established safety profile from over four years of accumulated data across multiple studies, positions VYKAT XR as a game-changer in the treatment of PWS.

The most common adverse reactions (≥10% and 2% greater than placebo) included hypertrichosis, edema, hyperglycemia, and rash. To support patient access, Soleno has launched Soleno One, a comprehensive patient support program accessible through VykatXR.com. This proactive approach to commercialization suggests that Soleno has strategically prepared for this approval, which can lead to higher adoption rates and revenue.

The financial implications for Soleno Therapeutics are substantial. With no competing approved therapies for hyperphagia in PWS, Soleno faces little pricing pressure, allowing the company to command premium pricing typical for orphan drugs. This, combined with the broad patient population covered by the FDA label—adults and children 4 years and older—maximizes the therapeutic's potential market and revenue per patient.

The approval of VYKAT XR also positions Soleno Therapeutics as a leader in the rare disease treatment landscape. The company's transformation from a clinical-stage to a commercial-stage organization validates their development capabilities and regulatory strategy execution. This milestone is a testament to the power of persistence, science, and advocacy, as families and researchers have worked tirelessly towards a treatment option that truly addresses the complexities of PWS.

In conclusion, the FDA approval of VYKAT XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families. For the PWS community, this approval offers hope and a significant improvement in their quality of life. For Soleno Therapeutics, it marks a watershed moment, positioning the company as a pioneer in the rare disease treatment landscape with strategic advantages in market share and revenue growth.