Santhera and Takeda to collaborate over neuromuscular disease drug

The clinical development of the drug, which has been granted orphan drug designation in the US and Europe, is about to enter a phase III clinical study in Europe and will enter a phase III study in the US shortly thereafter.

Santhera, a pharmaceutical company from Switzerland, will conduct all clinical development for regulatory approval in Europe and in the US, and have retained the rights to market the drug in the US. Meanwhile, Japan’s Takeda will support the development work and has obtained an exclusive license to market Idebenone in the European Union.

Under the terms of the agreement Santhera will receive an upfront payment of Euro 5 million, and will then receive development milestones and royalties from Takeda.

“I am pleased to announce Santhera’s first development and marketing partnership in this area with a company of the stature of Takeda,” said Dr Klaus Schollmeier, CEO of Santhera. “Santhera has already built significant R&D expertise in neuromuscular diseases. With the help of Takeda’s European organization, we are now leveraging that strength to bring our lead product, a decisive therapy breakthrough, to patients.”

Friedreich’s Ataxia severely impacts quality of life and reduces patients’ life expectancies substantially. Although the disease is primarily a neurological disease, cardiac symptoms are well recognized to be a life-threatening complication of the disease. The most common cardiac manifestation is hypertrophic cardiomyopathy which ultimately causes death in the majority of FRDA patients.