LoA Update: Agios’ Phase III mitapivat sulphate for pyruvate kinase deficiency sees 12-point rise in US approval prospects

Jennifer Smith 5th February 2021 (Last Updated February 5th, 2021 09:36)

As of 28 January, the likelihood of approval (LoA) by the FDA for Agios Pharmaceuticals’ Phase III drug for pyruvate kinase (PK) deficiency increased 12 points, according to GlobalData’s LoA data.

LoA Update: Agios’ Phase III mitapivat sulphate for pyruvate kinase deficiency sees 12-point rise in US approval prospects
The score change was based on results from the ACTIVATE-T trial of mitapivat sulphate in regularly transfused adults with PK deficiency. Credit: donfiore/Shutterstock.com.

As of 28 January, the likelihood of approval (LoA) by the FDA for Agios Pharmaceuticals’ Phase III drug for pyruvate kinase (PK) deficiency increased 12 points, according to GlobalData’s LoA data. The score change was based on results from the ACTIVATE-T trial of mitapivat sulphate in regularly transfused adults with PK deficiency.

On 26 January, the company announced ACTIVATE-T demonstrated a statistically significant in transfusion burden. Of the 27 patients enrolled in the trial, all of whom were given mitapivat, 37% had a ≥ 33% reduction in transfusion burden during the 24-week fixed dose period compared to individual historical transfusion burden standardised to 24 weeks (1-sided p=0.0002). In addition, 22% were transfusion-free during the 24-week fixed dose period. Agios states that it expects to file for regulatory approval, which will also include the previous ACTIVATE study, in the US in 2Q and in the EU in mid-2021, with a potential 2022 commercial launch in both geographies. Peak sales are expected to be $786m by 2026, according to GlobalData’s consensus forecasts. The company’s market cap is $3.6bn.

While the LoA prior to this news was 45%, GlobalData’s analysis using a combination of machine learning and a proprietary algorithm has notched up the LoA to 57%. In May 2020, this news service reported that experts had mixed views on the potential for Phase III success. The divide stemmed from differences in trial designs and populations between the Phase III program and the Phase II trial. Yet, some interviewed experts noted that even though the Phase II DRIVE-PK trial measured efficacy through secondary endpoints, the results were clinically meaningful and would bode well for the Phase III program.

by Jennifer C. Smith-Parker is Senior Editor  for Pharmaceutical Technology parent company GlobalData’s investigative journalism team. A version of this article originally appeared on the Insights module of GlobalData’s Pharmaceutical Intelligence Center. To access more articles like this, visit GlobalData.