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Agios’ Pyrukynd (mitapivat) Receives EMA's CHMP Positive Opinion Recommending the Marketing Authorization for the Treatment of PK Deficiency

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Agios’ Pyrukynd (mitapivat) Receives EMA's CHMP Positive Opinion Recommending the Marketing Authorization for the Treatment of PK Deficiency

Shots:

  • The CHMP opinion was based on the P-III (ACTIVATE) & (ACTIVATE-T) studies evaluating Pyrukynd in patients with PK deficiency. The EC’s decision was expected within 67 Days
  • Both the trials met their 1EPs i.e., patients achieved a hemoglobin response (40% vs 0%) over PBO in (ACTIVATE) trial & significant improvements for all pre-specified 2EPs incl. markers of hemolysis & ineffective erythropoiesis. In (ACTIVATE-T) trial, 37% achieved a ≥33% reduction in transfusion burden @24wk. fixed dose period, 22% were transfusion-free
  • If EC approved Pyrukynd, it will be 1st approved disease-modifying therapy for EU patients. The company also continues to advance the therapy in the 2 P-III trials for thalassemia & P-II/III (RISE UP) study in SCD

Ref: Globenewswire| Image: Agios

Click here to­ read the full press release 

Senior Editor

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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