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news.The European Commission (EC) has adopted a positive decision for designating Agios Pharmaceuticals’ oral, small molecule pyruvate kinase (PK) activator, mitapivat, as an orphan medicinal product (OMP) for treating sickle cell disease.
The oral, PK activator is intended to treat sickle cell disease. Credit: danilo.alvesd on Unsplash.
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This follows orphan drug status granted by the US Food and Drug Administration (FDA) in November 2020.
Currently, mitapivat is approved in the US under the brand name PYRUKYND for the treatment of haemolytic anaemia in adult patients with PK deficiency and also, for the same condition in the adult population within the European Union.
The therapy’s safety and efficacy are currently being evaluated through the RISE UP Phase II and Phase III studies.
These studies are part of a seamless Phase II/III protocol and are assessing the therapy’s impact on sickle cell subjects who have experienced two to ten sickle cell pain crises in the preceding 12 months and also have haemoglobin levels ranging from 5.5 to 10.5 g/dL while screening.
By enrolling various subjects but using the same sites, vendors, and resources, the two studies have managed to achieve operational efficiency.
Agios Pharmaceuticals R&D head and chief medical officer Sarah Gheuens said: “Alongside the 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.
“With the trial fully enrolled, we look forward to sharing the results of our Phase III RISE UP study evaluating the efficacy and safety of mitapivat in sickle cell disease with the community in late 2025.”
The OMP designation is intended to facilitate the development of treatments by offering incentives such as reduced fees and a ten-year period of market exclusivity.
