SRK-015, an experimental drug for SMA being developed by the US biotechnology company Scholar Rock, has been awarded the Orphan Medicinal Product designation in the European Union.
The designation carries incentives for pharmaceutical companies to encourage them to invest in developing treatments for orphan diseases.
SRK-015 is an experimental drug that aims to increase muscle reactivity in situations of lowered nerve impulses, as is usually the case in SMA. The molecule has been developed in close collaboration with the US SMA organisation CureSMA. It has already received orphan designations in the US and Japan.
Notwithstanding the fact that there is already a treatment for SMA – Spinraza – the European SMA community believes that we are still far from addressing the therapeutic needs on our continent, with the vast majority of SMA patients still unable to access any treatment. Consequently, we share the view that all drugs that aim to treat SMA should be awarded the EU orphan designation.