Tessa Romero

Alec Stranahan

Andrew Berens

Divya Rao

Gregory Renza

Christopher Raymond

Emily Bodnar

Eric Schmidt

Gregory Harrison

Lydia Erdman

Salveen Richter

Alex Nackenoff

Hiro Nagayumi

Imogen Mansfield

Marc Frahm

Matthew Guggenbiller

Theresa Robb

- **Agios Pharmaceuticals** received a **positive CHMP opinion** for **PYRUKYND® (Mitapivat)** for the treatment of anemia in adult patients with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia.  **[1439222_af7a6b02ec8248baa3dc249c660ea4c0_0]** 
- The **European Commission** is expected to make a **final decision by early 2026** regarding this new indication.  **[1439222_af7a6b02ec8248baa3dc249c660ea4c0_0]** 
- This positive opinion is based on the results from the global, randomized, double-blind, placebo-controlled Phase 3 studies **ENERGIZE and ENERGIZE-T**.  **[1439222_af7a6b02ec8248baa3dc249c660ea4c0_0]** 
- **Avanzanite Bioscience** entered an **exclusive partnership with Agios in June 2025** for the commercialization and distribution of PYRUKYND in the European Economic Area, UK, and Switzerland.  **[1439222_af7a6b02ec8248baa3dc249c660ea4c0_0]**

Agios Pharmaceuticals Receives Positive CHMP Opinion for PYRUKYND in Thalassemia

- Agios's partner, Avanzanite Bioscience, announced that the **Committee for Medicinal Products for Human Use (CHMP)** adopted a positive opinion for a new indication of **mitapivat** for the treatment of transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in adults.  **[1439222_6fead117ab31426d9f0e5746a58cdb51_0]** 
- Avanzanite entered an exclusive partnership with Agios in **June 2025** to manage the commercialization and distribution of mitapivat across Europe.  **[1439222_6fead117ab31426d9f0e5746a58cdb51_0]** 
- A final decision from the European Commission on this approval is anticipated in **early 2026**, which would mark thalassemia as mitapivat's second indication in Europe.  **[1439222_6fead117ab31426d9f0e5746a58cdb51_0]**

Agios's Partner Avanzanite Bioscience Announces Positive CHMP Opinion for Mitapivat in Thalassemia

- **Agios Pharmaceuticals** announced that the **Committee for Medicinal Products for Human Use (CHMP)** of the European Medicines Agency (EMA) adopted a **positive opinion for PYRUKYND® (mitapivat)** for the treatment of anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia in adults.  **[1439222_942d07ecd46e4bdd8650572da3bdbefd_0]** 
- This positive opinion, based on results from the **ENERGIZE-T and ENERGIZE Phase 3 trials**, means the **European Commission** will now review the CHMP's opinion, with a **final decision expected by early 2026**.  **[1439222_942d07ecd46e4bdd8650572da3bdbefd_0]** 
- In **June 2025**, Agios entered an exclusive agreement with **Avanzanite Bioscience B.V.** for the commercialization and distribution of PYRUKYND across the European Economic Area, the United Kingdom, and Switzerland, pending approval.  **[1439222_942d07ecd46e4bdd8650572da3bdbefd_0]** 
- PYRUKYND has also received approval in **Saudi Arabia** for thalassemia and is currently under review by health authorities in the **U.S.**, with a Prescription Drug User Fee Act (PDUFA) goal date of **December 7, 2025**, as well as in the **United Arab Emirates**.  **[1439222_942d07ecd46e4bdd8650572da3bdbefd_0]**

Agios' PYRUKYND Receives Positive CHMP Opinion for Thalassemia

AGIOS PHARMACEUTICALS (AGIO)