Orphan Designation for Silence’s β-Thalassemia Drug


Silence Therapeutics’ lead candidate SLN124 has secured orphan drug designation in the European Union for the treatment of β-Thalassemia.

The designation to SLN124 was granted by the Committee for Orphan Medicinal Products (COMP), a committee of the European Medicines Agency (EMA).

COMP after reviewing an application filed by the UK-based RNA therapeutics company concluded that the drug will be of significant benefit to those affected by β-Thalassemia, which is known to be a chronic and potentially life-threatening condition.

The positive opinion of the committee has been adopted by the European Commission. The decision from the EMA committee follows positive feedback from the UK Medicines and Healthcare products Regulatory Agency (MHRA) Scientific Advice meeting in last June, said Silence Therapeutics.

The orphan designation enables SLN124 to benefit from expedited clinical development to go along with ten years of market exclusivity, if approved by the European drug regulator.

According to Silence Therapeutics, SLN124 has been proven to have reduced serum iron levels and modulate tissue iron distribution in rodent models for β-Thalassemia and hereditary hemochromatosis. Further, it said that the investigational drug represents a promising therapeutic candidate for treatment of patients with iron overload disorders such as β-Thalassemia, hereditary hemochromatosis and myelodysplastic syndrome (MDS).

Silence Therapeutics CEO David Horn Solomon said:

We are pleased to receive Orphan Drug Designation for our iron overload disorder candidate, SLN124, which will assist in expediting clinical development as we progress our Phase Ib trial planned to begin in H2 2019. We believe that this innovative product offers significant promise for patients with iron overload disorders such as β-Thalassemia, MDS and hereditary hemochromatosis and we look forward to rapidly advancing SLN124 through clinical development.

Last month, Silence Therapeutics said that it had added a new short interfering RNA (siRNA) asset to its pre-clinical pipeline for the potential treatment of cardiovascular disease.

As per the company, SLN360 silences apolipoprotein, a component of lipoprotein, which it says is a highly validated target based on extensive human genetic data. Elevated levels of it have been related to increased risk of cardiovascular disease, irrespective of additional risk factors, said Silence Therapeutics.