SOM Biotech has received a positive opinion from the European Medicines Agency’s Committee for Orphan Medical Products for SOM3355, a candidate therapy for Huntington’s disease (HD). The molecule already holds orphan drug designation in the US.
SOM3355 combines mild beta-blocking activity with VMAT1 and VMAT2 inhibition. It has shown encouraging results in proof-of-concept and phase 2b studies in HD, a neurodegenerative condition with complex and evolving symptoms.
The EMA’s orphan designation is granted when a product shows potential to offer significant benefit to patients with unmet medical needs. This benefit must be confirmed at the time of marketing authorisation, in line with EU legislation.
Silvia Panigone, CEO of SOM Biotech, said: “Such promise, which must be substantiated in the phase 3 clinical study, will resonate not only with patients in Europe but also worldwide. This recognition supports SOM Biotech’s mission to explore the full potential of SOM3355 and to bring a novel first-line treatment option to the HD patients in the EU and beyond.”
HD symptoms range from motor and behavioural disturbances to psychiatric complications. Current treatments often target individual symptoms, leading to polypharmacy, poor adherence and increased risk of adverse events.
Rossella Medori, MD, CMO of SOM Biotech, said: “The dynamic nature of HD requires that more medications are prescribed based on disease progression. The need to improve quality of care by treating HD in its complexity is now well recognised. If the clinical profile of SOM3355 is confirmed, we hope to offer a streamlined medicinal intervention addressing multiple symptoms with good tolerability that is accepted well by individuals suffering from HD.”
SOM Biotech plans to advance SOM3355 through final development stages globally.
