Agios Seeks FDA Nod to Expand Mitapivat Label in Sickle Cell Disease

AGIO seeks FDA accelerated approval for mitapivat in sickle cell disease after mixed phase III data and a planned confirmatory study.

Zacks Investment Research • May 13, 2026

Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease

sNDA submission follows agreement with FDA on confirmatory trial, a requirement of the accelerated approval pathway Confirmatory trial designed to demonstrate clinical benefit of mitapivat on reducing transfusion burden in sickle cell disease CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the U.S. accelerated approval of mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease.

GlobeNewsWire • May 12, 2026

Agios to Present New Data at EHA 2026 Reinforcing the Significant Therapeutic Impact of Mitapivat Across Multiple Rare Hemolytic Anemias

Detailed results from RISE UP Phase 3 trial of mitapivat in sickle cell disease selected for distinguished Plenary Abstracts Session Company to host investor conference call and webcast during EHA 2026 on Saturday, June 13, at 9:00 a.m. ET (3:00 p.m.

GlobeNewsWire • May 12, 2026

AGIO Beats on Q1 Earnings & Sales, Stock Up 13% on New Drug Momentum

Agios shares jump 13% as Q1 revenues surge 138% on Aqvesme launch and Pyrukynd growth, while pipeline advances and SCD filing plans take shape.

Zacks Investment Research • Apr 30, 2026

Agios Pharmaceuticals, Inc. (AGIO) Q1 2026 Earnings Call Transcript

Agios Pharmaceuticals, Inc. (AGIO) Q1 2026 Earnings Call Transcript

Seeking Alpha • Apr 29, 2026

Agios Reports First Quarter 2026 Financial Results and Provides Business Update

Mitapivat (PYRUKYND® and AQVESME™) generated worldwide net revenues of $20.7 million in the first quarter of 2026, compared to $8.7 million in the first quarter of 2025 Strong initial U.S. commercial launch of AQVESME in thalassemia, with 242 prescriptions written as of March 31, 2026 Company plans to submit mitapivat sNDA for sickle cell disease in the second quarter of 2026 Pipeline advancing to multiple value-driving inflection points in 2026, including two Phase 2 readouts for next-generation PK activator tebapivat $1.0 billion in cash, cash equivalents, and marketable securities as of March 31, 2026 CAMBRIDGE, Mass., April 29, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced financial results and updates for the first quarter ended March 31, 2026.

GlobeNewsWire • Apr 29, 2026

Agios Pharmaceuticals (AGIO) Reports Q1 Loss, Tops Revenue Estimates

Agios Pharmaceuticals (AGIO) came out with a quarterly loss of $1.69 per share versus the Zacks Consensus Estimate of a loss of $1.81. This compares to a loss of $1.55 per share a year ago.

Zacks Investment Research • Apr 29, 2026

Earnings Preview: Agios Pharmaceuticals (AGIO) Q1 Earnings Expected to Decline

Agios Pharmaceuticals (AGIO) doesn't possess the right combination of the two key ingredients for a likely earnings beat in its upcoming report. Get prepared with the key expectations.

Zacks Investment Research • Apr 22, 2026

Agios Stock Sinks 23% as NVO's PKR Activator Meets Goal in SCD Study

AGIO shares plunge after Novo Nordisk's strong sickle cell data raised doubts about mitapivat's competitiveness and approval prospects.

Zacks Investment Research • Apr 21, 2026

Here's Why Agios Pharmaceuticals Crashed 23% Today

Agios Pharmaceuticals shares dropped 23% following Novo Nordisk's trial results. Novo's etavopivat outperformed Agios' mitapavit in key sickle cell endpoints, Novo plans to file for FDA approval in late 2026, threatening Agios' market potential.

The Motley Fool • Apr 20, 2026