15th Jan 2019 11:20
LONDON (Alliance News) - Silence Therapeutics on Tuesday said its SLN124 main candidate drug has been designated as an orphan drug in Europe for the treatment of beta-thalassemia.
Shares in the drug developer were up 7.4% at 56.80 pence on Tuesday.
A committee of the European Medicines Agency granted the designation, which means that clinical development of SLN124 will be expediated and the drug will be granted ten years of market exclusivity.
Beta-thalassemia is a blood disorder which results in a lack of oxygen in parts of a patient's body, as well as a shortage of red blood cells.
Patients with more serious forms of beta-thalassemia require many blood transfusions to boost their red blood cell numbers. However, a side effect of these transfusions is that they can cause iron to build up in the body to a harmful degree.
In rodent models, SLN124 has been shown to lower serum iron levels and control the distribution of iron into the body's tissues.
SLN124 is also a promising candidate for treating other iron overload disorders.
The orphan drug designation is expected to assist with the progression of Slience's phase 1b trial, which is scheduled to begin in the second half of 2019.
"We believe that this innovative product offers significant promise for patients with iron overload disorders ... and we look forward to rapidly advancing SLN124 through clinical development," said Silence Chief Executive David Horn Solomon.
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