Zatolmilast Receives Orphan Medicinal Product Designation from the European Commission: Promising First-in-Class Therapy for Fragile X Syndrome

Shionogi B.V., the European arm of Shionogi & Co., Ltd., has revealed that the European Commission has granted Orphan Medicinal Product designation to zatolmilast (BPN14770), an investigational treatment for Fragile X syndrome (FXS), a prominent cause of inherited intellectual disability and autism. This designation is reserved for therapies that could offer significant benefits for conditions affecting fewer than 5 in 10,000 individuals in Europe, acknowledging the substantial impact rare diseases like FXS have on affected individuals who often face challenges in accessing diagnosis, treatment, and research opportunities.

FXS is a neurodevelopmental disorder characterized by a spectrum of cognitive, behavioral, and physical difficulties, including limitations in self-care and communication abilities. Despite its profound effects, there are currently no pharmacological treatments specifically approved by the European Medicines Agency for FXS, underscoring the urgent need for innovative therapeutic options.

Shionogi is committed to addressing unmet medical needs, particularly for patients with limited treatment options. In line with this commitment, Shionogi acquired Tetra Therapeutics, the developer of zatolmilast, in 2020. Zatolmilast, if approved, aims to be the first cognitive treatment designed specifically for FXS.

Pete Richardson, Managing Director of the UK Fragile X Society, emphasized the challenges individuals and families face in accessing adequate support and expressed optimism about the potential of zatolmilast to improve the well-being of those living with FXS.

Juan-Carlos Gomez, MD, Chief Medical Officer at Shionogi, highlighted the company’s dedication to developing innovative medicines and welcomed the regulatory recognition of zatolmilast’s clinical potential in Europe.

FXS is a leading genetic cause of inherited intellectual disability and autism, affecting males more severely due to the mutation of the Fragile X Messenger Ribonucleoprotein 1 (FMR1) gene on the X chromosome. Common symptoms include global developmental delay, intellectual disability, behavioral issues, attention deficits, and anxiety, impacting various aspects of daily life.

Tetra Therapeutics, a subsidiary of the Shionogi Group, focuses on developing therapeutic products for central nervous system diseases and disorders, with zatolmilast as a key component of its clinical program. Additionally, Tetra is engaged in pre-clinical development of a PDE4B Inhibitor.

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