Logo

Agios’ Pyrukynd (mitapivat) Receives the US FDA’s Approval as First Disease-Modifying Therapy for Hemolytic Anemia in Adults with PK Deficiency

Share this

Agios’ Pyrukynd (mitapivat) Receives the US FDA’s Approval as First Disease-Modifying Therapy for Hemolytic Anemia in Adults with PK Deficiency

Shots:

  • The approval was based on the P-III (ACTIVATE) & (ACTIVATE-T) studies to evaluate Pyrukynd vs PBO in patients with hemolytic anemia with PK deficiency. The therapy is expected to be available in the US in ~2wks.
  • Both trials met their 1EPs i.e., In (ACTIVATE) trial, patients achieved a hemoglobin response (40% vs 0%), improvements for all pre-specified 2EPs including markers of hemolysis & ineffective erythropoiesis, changes in jaundice, tiredness & shortness of breath
  • In (ACTIVATE-T) trial, 33% achieved a transfusion reduction, 22% were transfusion-free. The therapy is also under EMA’s review for PK deficiency while the regulatory decision in the EU is expected at end of 2022

 Ref: Globe Newswire | Image: BioSpace

Click here to­ read the full press release 

Senior Editor

This content piece was prepared by our former Senior Editor. She had expertise in life science research and was an avid reader. For any query reach out to us at connect@pharmashots.com

Share this article on WhatsApp, LinkedIn and Twitter

Join the PharmaShots family of 12000+ subscribers

I accept the Terms and Conditions