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Agios Pharmaceuticals, Inc, a leader in cellular metabolism and pyruvate kinase activation pioneering therapies for rare diseases, announced that the European Commission has adopted a positive decision for the designation of Mitapivat, an oral, small molecule pyruvate kinase activator, as an orphan medicinal product for the treatment of sickle cell disease.
Earlier, in November 2020, the US Food and Drug Administration (FDA) also granted orphan drug designation to Mitapivat for sickle cell disease.
“Alongside the US FDA’s orphan drug designation in the US, the European Commission’s orphan medicinal product designation for Mitapivat underscores the urgent need for novel therapies for sickle cell disease and highlights its potential to provide clinically meaningful benefits to patients navigating this debilitating condition,” said Sarah Gheuens, chief medical officer and head of R&D at Agios Pharmaceuticals. “With the trial fully enrolled, we look forward to sharing the results of phase 3 RISE UP study evaluating the efficacy and safety of mitapivat in sickle cell disease with the community in late 2025.”
The European Commission offers orphan medicinal product designation to innovative therapies that address life-threatening or chronically debilitating conditions affecting fewer than five in 10,000 individuals in the European Union, and that have the potential to provide a significant benefit over existing treatments. This designation provides extensive benefits to encourage the development of these medicines, including reduced fees and a 10-year period of market exclusivity.
The RISE UP phase 2 and phase 3 studies are evaluating the efficacy and safety of Mitapivat in sickle cell disease patients who are 16 years of age or older, have had between two and 10 sickle cell pain crises in the past 12 months, and have hemoglobin within the range of 5.5 to 10.5 g/dL during screening. The phase 2 and phase 3 studies are conducted under a single operationally seamless phase 2/3 protocol. The two studies enrolled different participants and achieved operational efficiency through leveraging the same sites, vendors and other resources.
The phase 2 study included a 12 weeks randomized, placebo-controlled period in which participants were randomized in a 1:1:1 ratio to receive 50 mg Mitapivat twice daily, 100 mg Mitapivat twice daily or matched placebo. The primary endpoints were hemoglobin response, defined as =1.0 g/dL increase in average hemoglobin concentration from week 10 through week 12 compared to baseline, and safety.
The phase 3 study includes a 52 weeks randomized, placebo-controlled period in which participants will be randomized in a 2:1 ratio to receive 100 mg of Mitapivat twice daily or matched placebo. The primary endpoints are hemoglobin response, defined as a =1.0 g/dL increase in average hemoglobin concentration from week 24 through week 52 compared with baseline, and annualized rate of sickle cell pain crises. In October 2024, Agios announced that enrollment in the phase 3 study had been completed, with more than 200 patients enrolled worldwide.
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