Rocket Pharmaceuticals to Participate in the 44th Annual J.P. Morgan Healthcare Conference

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today ...

Agios Pharma shares jump as U.S. FDA expands approval for blood disorder drug

Agios Pharma's shares soar 16% after FDA expands approval for its blood disorder drug, Aqvesme, set for late next month.
Pharmabiz

Agios Pharma’s oral PK activator, Aqvesme receives US FDA approval to treat anaemia in adults with alpha- or beta-thalassemia

Agios Pharma’s oral PK activator, Aqvesme receives US FDA approval to treat anaemia in adults with alpha- or beta-thalassemia: Cambridge, Massachusetts Friday, December 26, 2025 ...

Here's why shares in Agios Pharmaceuticals popped today

The company received FDA approval for its leading drug, and investors were pleased with the update from management.

AGIO's Mitapivat Gets FDA Nod for Thalassemia Indication with REMS in Label

Agios Pharmaceuticals wins FDA approval for Aqvesme in alpha- and beta-thalassemia, making mitapivat the only approved option, despite a new REMS.

Agios Pharmaceuticals stock soars after FDA approves AQVESME for thalassemia

The approval makes AQVESME the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, a rare blood disorder. The drug is expected ...
MedPage Today on MSN

FDA approves mitapivat for thalassemia-related anemia

An oral pyruvate kinase (PK) activator, mitapivat becomes the only approved option for treating anemia in both non-transfusion- and transfusion-dependent patients with alpha- or beta-thalassemia, ...

U.S. FDA Approves Agios’ AQVESME™ (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

Detailed price information for Agios Pharmaceuticals (AGIO-Q) from The Globe and Mail including charting and trades.
Fox News

Nutrient deficiency linked to heart disease risk for millions, new study warns

More than three-quarters of the global population is falling short on omega-3 intake, a nutrient gap that may increase the risk of heart disease, cognitive decline, inflammation and vision problems.
MedCity News

UCB Drug Becomes First FDA-Approved Treatment for Ultra-Rare and Fatal Mitochondrial Disease

The energy that powers cells of the human body comes from tiny cellular components called mitochondria. Just as a draining battery makes it difficult to operate an electronic device, mitochondrial ...
GEN

FDA Approves First Treatment for Rare Thymidine Kinase 2 Deficiency

Thymidine kinase 2 deficiency (TK2d) is an ultra-rare (1.64 cases per 1,000,000 people) often fatal, genetic mitochondrial disease characterized by progressive and severe muscle weakness. Those ...
Monthly Prescribing Reference

FDA Approves Kygevvi for Thymidine Kinase 2 Deficiency

Kygevvi is expected to be commercially available in the US in the first quarter of 2026. The Food and Drug Administration (FDA) has approved Kygevvi ® for the treatment of thymidine kinase 2 ...