Barcelona-based biotech advances Huntington’s programme as EMA backs orphan designation, reinforcing regulatory momentum already gained in the US.

SOM Biotech has received a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) on granting Orphan Drug Designation (ODD) for its lead candidate SOM3355 in Huntington’s disease (HD).

The Barcelona-based clinical-stage company focuses on rare central nervous system disorders and has positioned SOM3355 as a novel therapeutic option with a multimodal mechanism of action. The molecule is a mild beta-blocker as well as an inhibitor of VMAT1 and VMAT2, targeting multiple aspects of HD’s complex symptomatology.

The candidate already holds orphan designation in the United States and has demonstrated efficacy in proof-of-concept and Phase 2b clinical studies. The EU opinion provides further regulatory validation ahead of Phase 3 development, while offering SOM Biotech access to incentives including protocol assistance, fee reductions, and potential market exclusivity.

Silvia Panigone, CEO of SOM Biotech, said the 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,” while emphasising that the promise “must be substantiated in the phase 3 clinical study.”

Current treatment options for Huntington’s disease are limited and typically address individual symptoms such as motor, behavioural, or psychiatric disturbances. Dopamine depleting agents remain standard of care, but their side effects can overlap with disease progression, contributing to polypharmacy, poor adherence, and increased hospitalisations.

Rossella Medori, CMO of SOM Biotech, said that “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.”

The company said it remains fully committed to progressing SOM3355 into the final stage of global development. A pivotal Phase 3 trial is expected to define whether the candidate can deliver on its promise to reshape HD management, and potentially reduce the need for polypharmacy in this challenging rare disease.