The U.S. Food and Drug Administration approved two new therapies in late December, authorizing Agios Pharmaceuticals’ oral drug Aqvesme for the treatment of anemia in adults with alpha- or beta-thalassemia on Dec. 23, followed by approval of Vanda Pharmaceuticals’ motion sickness drug Nereus on Dec. 30.
The FDA’s decision on Aqvesme followed multiple review delays. The agency moved its original decision date from Sept. 7 to Dec. 7 after determining it needed additional time to evaluate the drug’s Risk Evaluation and Mitigation Strategy (REMS), which was designed to address the risk of liver injury associated with the treatment. In early December, Agios said the review timeline was extended again as the company worked with the FDA to finalize labeling documents connected to the REMS program.
Aqvesme, which contains the active ingredient mitapivat, is taken twice daily and is approved for adults with either transfusion-dependent or non-transfusion-dependent alpha- or beta-thalassemia. The approval expands the use of mitapivat beyond its earlier authorization. Nearly four years ago, the FDA approved the drug under the brand name Pyrukynd to reduce hemolytic anemia in adults with pyruvate kinase deficiency.
Agios stated that the approval allows treatment to be extended to a broader thalassemia patient population. The therapy relieves patients from the burden of transfusions or subcutaneous treatments. During a Christmas Eve conference call, Agios Chief Executive Officer Brian Goff said Aqvesme is the “first medicine to show quality of life improvements in non-transfusion-dependent patients, which we understand to be of critical importance.”
The Aqvesme label includes a boxed warning for hepatocellular injury. As part of the REMS program, patients are required to undergo liver testing prior to starting therapy and then every four weeks for the first 24 weeks after treatment initiation. Agios said Aqvesme will be made available in late January following implementation of the REMS program.
The company set the annual price at $425,000, compared with the $335,000 wholesale acquisition cost of Pyrukynd. Agios said the use of separate brand names allows the REMS program to apply only to Aqvesme and not to Pyrukynd.
Agios reported Pyrukynd sales of $34 million during the first three quarters of 2025. The company said 149 patients were receiving the therapy in the third quarter, compared with 136 patients in the first quarter. Agios estimates that 4,000 patients in the United States fall within the addressable thalassemia population.
Vanda’s approval for Nereus followed the FDA’s decision earlier in December to lift a partial clinical hold. The agency concluded that motion sickness is not a chronic condition and determined that Nereus is an acute, event-driven therapy. Based on that conclusion, the FDA found that a six-month toxicity study was not required.
Nereus, also known as tradipitant, is approved to reduce the incidence of motion-induced vomiting. The approval was supported by three phase 3 trials, including two conducted on boats. In one study involving 365 participants, 19% of those receiving Nereus experienced vomiting, compared with 44% of participants receiving a placebo. Vanda said the drug will be made available in the coming months and stated that it is the first new pharmacologic treatment for motion sickness in more than four decades.
The approval comes 15 months after the FDA rejected tradipitant for the treatment of gastroparesis. That decision prompted Vanda to criticize the agency for delays in the review process and for not convening an advisory committee meeting. Vanda said it continues to pursue approval of tradipitant for gastroparesis and is also studying the drug for nausea and vomiting associated with GLP-1 obesity treatments.
Nereus becomes the fourth marketed drug for Vanda, which previously received approval for the schizophrenia drug Fanapt in 2009 and later expanded its use to treat bipolar I disorder in 2024. Vanda also sells sleep disorder drug Hetlioz and multiple sclerosis treatment Ponvory. The FDA is scheduled to decide on Hetlioz as a treatment for jet lag disorder on Jan. 7.
