FDA Approves New Treatments for Thalassemia and Motion Sickness

The U.S. Food and Drug Administration (FDA) has ended 2025 with two significant approvals, granting the green light to Agios Pharmaceuticals' Aqvesme for thalassemia and Vanda Pharmaceuticals' Nereus for motion sickness. These approvals mark important milestones in their respective therapeutic areas, offering new options for patients and expanding the companies' product portfolios.

Agios' Aqvesme Breaks New Ground in Thalassemia Treatment

On December 23, 2025, the FDA approved Agios' oral medicine Aqvesme (mitapivat) for the treatment of anemia in adults with alpha- or beta-thalassemia. This approval comes after a series of regulatory hurdles, including decision date shifts and delays related to the finalization of labeling documents for the drug's Risk Evaluation and Mitigation Strategy (REMS).

Aqvesme represents a significant advancement in thalassemia treatment, offering patients an alternative to transfusions or subcutaneous treatments. Brian Goff, CEO of Agios, highlighted the drug's impact, stating, "It is the first medicine to show quality of life improvements in non-transfusion-dependent patients, which we understand to be of critical importance."

The approval of Aqvesme opens up treatment to a larger patient population, including both non-transfusion-dependent and transfusion-dependent individuals with alpha- or beta-thalassemia. Agios plans to make Aqvesme available in late January 2026, following the implementation of its REMS program. The company has set the annual cost of Aqvesme at $425,000, a significant increase from the $335,000 wholesale acquisition cost of its predecessor, Pyrukynd.

Importantly, Aqvesme's label will include a boxed warning for hepatocellular injury. The REMS program mandates liver testing for patients before initiating treatment and every four weeks for the first 24 weeks of use.

Vanda's Nereus Offers New Hope for Motion Sickness Sufferers

On December 30, 2025, the FDA approved Vanda Pharmaceuticals' Nereus (tradipitant) for reducing the incidence of motion-induced vomiting. This approval marks a significant milestone, as Nereus becomes the first new pharmacologic treatment for motion sickness in over four decades.

The journey to approval for Nereus was not without challenges. The FDA had previously rejected the drug for treating nausea and vomiting in patients with gastroparesis. However, after lifting a partial clinical hold on December 4, 2025, the agency concluded that motion sickness is not a chronic condition and that Nereus is an "acute, event-driven therapy."

Nereus' approval was supported by three phase 3 trials, including two conducted on boats. In one study involving 365 participants, Nereus demonstrated a 19% incidence of vomiting compared to a 44% incidence for those on placebo.

Vanda plans to make Nereus available in the "coming months," adding it to their existing portfolio of four marketed drugs. The company continues to explore additional indications for tradipitant, including its potential use in preventing nausea and vomiting induced by GLP-1 obesity drugs.

These recent FDA approvals underscore the ongoing innovation in the pharmaceutical industry, providing new treatment options for patients with thalassemia and motion sickness. As these drugs enter the market, their impact on patient care and company revenues will be closely watched by industry observers.