Agios Receives FDA Approval for AQVESME to Treat Thalassemia

Written by Emily J. Thompson, Senior Investment Analyst

AGIO-2.47%

Source: Newsfilter

Updated: Dec 26 2025

0mins

Source: Newsfilter

Exclusive Approval: AQVESME becomes the only FDA-approved treatment for both non-transfusion-dependent and transfusion-dependent thalassemia, marking a significant breakthrough in treatment options for this severe disease and is expected to greatly improve patients' quality of life.
Clinical Trial Success: In the global randomized double-blind ENERGIZE and ENERGIZE-T Phase 3 trials, 452 patients participated, demonstrating that AQVESME significantly outperformed placebo in improving anemia and fatigue, meeting all primary and key secondary efficacy endpoints.
Market Outlook: AQVESME is expected to be available in late January 2026, providing a new treatment option for approximately 6,000 adult thalassemia patients in the U.S., filling a critical gap in the market.
Investor Conference: Agios will host an investor conference call on December 24, 2025, to discuss the FDA approval of AQVESME, further enhancing investor confidence in the company's future growth.

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AGIO

$27.61+Infinity%1D

Analyst Views on AGIO

Wall Street analysts forecast AGIO stock price to rise over the next 12 months. According to Wall Street analysts, the average 1-year price target for AGIO is 36.38 USD with a low forecast of 25.00 USD and a high forecast of 62.00 USD. However, analyst price targets are subjective and often lag stock prices, so investors should focus on the objective reasons behind analyst rating changes, which better reflect the company's fundamentals.

9 Analyst Rating

6 Buy

3 Hold

0 Sell

Moderate Buy

Current: 28.310

Low

25.00

Averages

36.38

High

62.00

Current: 28.310

Low

25.00

Averages

36.38

High

62.00

JPMorgan

Neutral

maintain

$20 -> $25

2026-01-06

Reason

JPMorgan

Price Target

$20 -> $25

AI Analysis

2026-01-06

maintain

Neutral

Reason

JPMorgan raised the firm's price target on Agios Pharmaceuticals to $25 from $20 and keeps a Neutral rating on the shares. The firm updated the company's model post the approval of Aqvesme.

BofA

Buy

maintain

$34 -> $35

2026-01-06

Reason

BofA

Price Target

$34 -> $35

2026-01-06

maintain

Buy

Reason

BofA raised the firm's price target on Agios Pharmaceuticals to $35 from $34 and keeps a Buy rating on the shares. The firm is updating its price targets for U.S Biopharmaceuticals under its coverage, the analyst tells investors. Over the past few months, several key items "have fallen into place," including positive data catalysts being rewarded; large-cap biopharma spending cash on M&A and in-licensing; growing backlog of companies on the private side; improving access to capital; and limited impact from drug price regulation, BofA says. The firm believes biotech is back, but the biggest concern is whether this will last.

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About AGIO

Agios Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on developing and delivering transformative therapies for patients living with rare diseases. It markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for debilitating hemolytic anemia. Its lead product candidate in its portfolio, PYRUKYND (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. It is also developing tebapivat, a novel PK activator, for the potential treatment of lower-risk myelodysplastic syndromes, or LR MDS, and hemolytic anemias; AG-181, its phenylalanine hydroxylase, or PAH, stabilizer for the potential treatment of phenylketonuria, or PKU; and AG-236, an siRNA in-licensed from Alnylam Pharmaceuticals, Inc., targeting the transmembrane serine protease 6, or TMPRSS6 gene for the potential treatment of polycythemia vera, or PV.

About the author

Emily J. Thompson

Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.