Drug Discovery and Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50

Mitapivat from Agios scores FDA nod as for hemolytic anemia in adults

By Brian Buntz | February 18, 2022

AgiosAgios Pharmaceuticals Inc. (NASDAQ:AGIO) has announced that Pyrukynd (mitapivat) has won FDA approval to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

A first-in-class oral PK activator, mitapivat is the first disease-modifying therapy for hemolytic anemia to win FDA approval.

FDA based its decision on data from the Phase 3 ACTIVATE and ACTIVATE-T studies. Mitapivat met its primary endpoint in both studies.

Last year, Agios announced that 37% of patients who received mitapivat in the ACTIVATE trial achieved at least 33% reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks.

To maximize the availability of Pyrukynd, Agios is offering select patients $0 copays for the medication. The company also has a patient assistance program (PAP) that provides free prescriptions to certain uninsured and underinsured patients.

“For more than a decade, we have been pioneering the science of PK activation in order to bring Pyrukynd to people with PK deficiency and provide them with the first medication approved specifically to address this rare, debilitating blood disorder,” Agios CEO Jackie Fouse said in a statement.

AGIO shares fell 5.18% to $29.46.


Filed Under: clinical trials, Drug Discovery, Hematology
Tagged With: Agios Pharmaceuticals, hemolytic anemia, PK deficiency, Pyrukynd, pyruvate kinase
 

Tell Us What You Think! Cancel reply

You must be logged in to post a comment.

Related Articles Read More >

Akebia/Otsuka
Akebia Therapeutics gets complete response letter from the FDA for vadadustat for CKD-associated anemia
Adakveo
Adakveo: An FDA-indicated treatment for sickle cell disease
xospata
Xospata met primary endpoint in confirmatory trial
FibroGen
FDA advisory committee recommends not approving FibroGen’s anemia drug candidate roxadustat

MEDTECH 100 INDEX

Medtech 100 logo
Market Summary > Current Price
The MedTech 100 is a financial index calculated using the BIG100 companies covered in Medical Design and Outsourcing.

Need Drug Discovery news in a minute?

We Deliver!
Drug Discovery & Development Enewsletters get you caught up on all the mission critical news you need. Sign up today.
Enews Signup
Drug Discovery and Development
  • MASSDEVICE
  • DEVICETALKS
  • Medical Design & Outsourcing
  • MEDICAL TUBING + EXTRUSION
  • MEDTECH 100
  • Medical Design Sourcing
  • Subscribe to our Free E-Newsletter
  • Contact Us
  • About Us
  • Advertise With Us
  • R&D World
  • Drug Delivery Business News
  • Pharmaceutical Processing World

Copyright © 2022 WTWH Media LLC. All Rights Reserved. The material on this site may not be reproduced, distributed, transmitted, cached or otherwise used, except with the prior written permission of WTWH Media
Privacy Policy | Advertising | About Us

Search Drug Discovery & Development

  • Home Drug Discovery and Development
  • Drug Discovery
  • Genomics/Proteomics
  • Oncology
  • Neurological Disease
  • Infectious Disease
  • R&D 100 Awards
  • Pharma 50
    • 2022 Pharma 50
    • 2021 Pharma 50