Agios’ Pyrukynd (mitapivat) Receives the EC’s Marketing Authorization for the Treatment of Pyruvate Kinase (PK) Deficiency
Shots:
- The marketing authorization was based on the (ACTIVATE) & (ACTIVATE-T) trials evaluating Pyrukynd in adults with PK deficiency
- Both the trial met its 1EPs i.e., In a trial (ACTIVATE), 40% achieved Hb response who are not regularly transfused, & significant improvements were also demonstrated for all pre-specified 2EPs incl. markers of hemolysis & ineffective erythropoiesis
- In the trial (ACTIVATE-T), 37% achieved a transfusion reduction response in the 24wk. fixed dose period over the historical transfusion burden standardized who are regularly transfused, 22% were transfusion-free. The company continues to advance the therapy in P-III (ENERGIZE) & (ENERGIZE-T) studies for thalassemia along with P-II/III (RISE UP) study in SCD
Ref: Globenewswire | Image: Agios
Click here to read the full press release
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.
