Agios scores FDA approval for PK deficiency drug Pyrukynd

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By: Anna Bratulic

Ref: The Boston Globe, FDA, GlobeNewswire, FDA, Agios Pharmaceuticals

Published: 02/17/2022

Agios scores FDA approval for PK deficiency drug Pyrukynd

The FDA on Thursday approved Agios Pharmaceuticals' Pyrukynd (mitapivat) to treat haemolytic anaemia in adults with pyruvate kinase (PK) deficiency, regardless of transfusion status. The company noted that the oral PK activator, which will carry an average annual wholesale acquisition cost of $334,880, is the first approved disease-modifying therapy for the rare blood condition.

The filing, granted a priority review by the FDA last year, was based on results from two Phase III trials. In the ACTIVATE study, Pyrukynd demonstrated a significant increase in haemoglobin (Hb) in patients with PK deficiency who are not regularly transfused, with 40% of participants achieving Hb response, compared to none in the placebo group. There were significant improvements on all pre-specified secondary endpoints as well, including markers of haemolysis and ineffective erythropoiesis.

Pyrukynd also succeeded in the ACTIVATE-T trial, demonstrating "significant and clinically meaningful" reductions in transfusion burden for patients who are regularly transfused. In the study, 33% of patients achieved a transfusion reduction response over a specific threshold in the 24-week fixed dose period compared to individual historical transfusion burden. Moreover, 22% were transfusion-free during the fixed-dose period.

Advancing thalassaemia, sickle cell disease trials

Agios CEO Jackie Fouse said "we remain committed to partnering with patients, caregivers, advocates and healthcare providers to ensure that the impact of Pyrukynd is maximised through robust support…and access programmes" aimed at reducing or eliminating out-of-pocket costs, including $0 copays and free medication for eligible patients. The company noted that it "is committed to not raise the price for five years." Agios submitted a marketing application for the drug to EU regulators last June seeking approval in the same indication.

Meanwhile, Agios expects to kick off two more studies of Pyrukynd in mid-2022, ACTIVATE-kids and ACTIVATE-kidsT, in paediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively.

Fouse said she hopes the treatment will eventually generate annual sales of more than $1 billion, assuming it is approved for two other blood disorders. The company said it is pushing ahead with a pair of Phase III thalassaemia trials, dubbed ENERGIZE and ENERGIZE-T, as well as its Phase II/III RISE UP study in sickle cell disease.