Syntara (ASX:SNT) has achieved a significant regulatory milestone, announcing it has received a positive opinion from the European Medicines Agency (EMA) on its application for Orphan Drug Designation (ODD) for amsulostat (SNT-5505) in myelofibrosis.
The designation, once formally granted, will provide substantial incentives to support the drug’s continued development in the European Union, including ten years of market exclusivity upon approval, protocol assistance for clinical trials, access to the centralised authorisation pathway, and reductions in key regulatory fees.
Amsulostat, Syntara’s lead clinical candidate, already holds ODD status in the United States, awarded by the Food and Drug Administration for the treatment of myelofibrosis.
Myelofibrosis is a severe bone marrow disorder in which healthy tissue is gradually replaced by fibrotic, scar-like material. This progressive damage leads to declining production of red and white blood cells and platelets, often resulting in profound fatigue, infections, bleeding complications, and splenomegaly. The condition typically emerges later in life, most commonly between the ages of 60 and 70, and its underlying cause remains poorly understood.
Syntara recently reported encouraging top-line Phase 2a data showing that amsulostat delivered substantial clinical improvements in patients inadequately controlled on ruxolitinib. In the study, 73 per cent of participants achieved at least a 50 per cent reduction in total symptom score, and nearly half experienced a meaningful reduction in spleen volume after one year of treatment. A favourable tolerability profile with no treatment-related adverse events identified supported these results.
The company has also received guidance from the FDA on its recommended pathway for further development and is now progressing the design of the next clinical trial for amsulostat.
Syntara CEO Gary Phillips said the positive opinion from the EMA provides further validation of the potential of amsulostat as a differentiated therapy in a challenging disease area.
“Receipt of this positive opinion for Orphan Drug Designation in the EU comes after detailed review of the amsulostat pre-clinical and clinical dossier by the EMA, providing further validation of the potential that the drug has to benefit myelofibrosis patients,” Phillips said. “We continue to engage with our advisors and regulatory authorities to design a clinical trial that leverages the product’s strong differentiating features as a well-tolerated and effective therapy. Our goal is to ensure the study design is compelling for investors and strategic partners by clearly demonstrating how amsulostat can positively impact the current standard of care."