The European Medicines Agency (EMA) has validated the marketing authorisation application (MAA) submitted by Soleno Therapeutics for diazoxide choline prolonged-release tablets, aimed at treating Prader-Willi syndrome in the adult and paediatric population aged four and above.
The application focuses on those individuals who have hyperphagia, a condition causing intense, persistent hunger.
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Soleno has secured orphan drug designation in the European Union (EU) for diazoxide choline, which could grant a market exclusivity of up to ten years upon approval, alongside other regulatory and financial benefits.
In March this year, the US Food and Drug Administration (FDA) approved the therapy under the brand name Vykat XR for treating extreme hunger in Prader-Willi syndrome patients. This came after a three-month postponement from the agency.
Prader-Willi syndrome is stated to be a rare genetic neurodevelopmental disorder stemming from an abnormality on chromosome 15 gene expression.
According to the company’s estimation, around 9,500 individuals in the UK, Germany, Italy, Spain, and France are affected by this syndrome.
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By GlobalDataHyperphagia, the syndrome’s hallmark, can lead to life-threatening situations and long-term health issues like obesity, cardiovascular disease, and diabetes.
The relentless hunger and associated behaviours significantly impact the quality of life for those with Prader-Willi syndrome and their families.
Soleno Therapeutics CEO and chairman Anish Bhatnagar said: “The validation of our MAA represents the next significant milestone in our mission to deliver this important therapy to the broad Prader-Willi syndrome community, including those in the EU.
“Based on the data generated, DCCR has the potential to help treat hyperphagia, which is the most life-limiting aspect of Prader-Willi syndrome. We look forward to working closely with European regulators during the review process and intend to make DCCR available to patients in the EU as expeditiously as possible, if approved.”
