The FDA delivered a pair of holiday season approvals a week apart, signing off on Agios’ oral medicine Aqvesme for the treatment of anemia in adults with alpha- or beta-thalassemia on Dec. 23, and then issuing its final new drug approval of the year to Vanda Pharmaceuticals for motion sickness pill Nereus on Dec. 30.
As has been the case with many FDA decisions in this year of cutbacks and leadership transition, neither nod was accomplished easily.
The endorsement for Agios came after the FDA shifted its decision date from Sept. 7 to Dec. 7, needing additional time to review the company’s Risk Evaluation and Mitigation Strategy (REMS) to mitigate potential liver injury that comes with the use of Aqvesme (mitapivat). Then, in early December, the company explained that there was another decision delay as it was working with the agency to “finalize the labeling documents” for its REMS plan.
As for Vanda, its approval arrived after the FDA lifted a partial clinical hold on Dec. 4, concluding that motion sickness is not a chronic condition and that Nereus (tradipitant) is an “acute, event-driven therapy.” With that, the U.S. regulator determined that a six-month toxicity study of tradipitant was not necessary.
Agios’ 2nd nod for mitapivat
This is not Agios’ first go-round with the FDA on mitapivat. Nearly four years ago, the agency blessed it with the commercial name Pyrukynd as the first treatment to reduce hemolytic anemia in adults with the rare, genetic blood disorder pyruvate kinase (PK) deficiency.
The endorsement for twice-daily Aqvesme opens the treatment up to a larger patient population, including those with non-transfusion-dependent or transfusion-dependent alpha- or beta-thalassemia.
It relieves patients from the burden of transfusions or subcutaneous treatments. It is also the “first medicine to show quality of life improvements in non-transfusion-dependent patients, which we understand to be of critical importance,” Agios CEO Brian Goff said in a Christmas Eve conference call.
The company will make Aqvesme available in late January after its REMS program is implemented, Agios said. It will charge $425,000 for a yearly supply of Aqvesme, which is up from the $335,000 wholesale acquisition cost of Pyrukynd.
The separate brand names for mitapivat allows Agios to “isolate the Aqvesme REMS to thalassemia, ensuring regulatory clarity and confirming that it does not apply to Pyrukynd for PK deficiency,” Goff added.
The label of Aqvesme will include a boxed warning for hepatocellular injury. The REMS program will require patients to undergo liver testing before beginning treatment and then every four weeks of the next 24 weeks after initiating its use.
Agios reported sales of Pyrukynd reached $34 million for the first three quarters of 2025, with 149 patients on therapy in the third quarter, up from 136 in the first quarter. The company estimates the addressable thalassemia patient population at 4,000 in the U.S.
Vanda scores with Nereus
The FDA motion sickness blessing for Nereus comes 15 months after the FDA rejected the oral neurokinin-1 (NK-1) receptor antagonist to reduce nausea and vomiting in patients with the digestive disorder gastroparesis.
The complete response letter (CRL) triggered a fiery response from the Washington, D.C.-based drugmaker, which blasted the agency for the delay in its decision and for not conducting an advisory committee meeting to discuss the treatment's value.
Nereus made the grade, however, as the FDA blessed it to reduce the incidence of motion-induced vomiting. With the nod, Nereus has become the first new pharmacologic treatment for the condition in more than four decades, the drugmaker said.
The approval was backed by three phase 3 trials, including two conducted on boats, which showed that it reduced nausea in participants with a history of motion sickness. In one boat study of 365 participants, there was a 19% incidence of vomiting of those on Nereus compared to a 44% incidence for those on placebo.
Vanda said it will make Nereus available in the “coming months.” It becomes the fourth drug on the market for Vanda, which scored a 2009 approval for schizophrenia treatment Fanapt, followed by a 2024 expansion to treat bipolar I disorder. The new indication helped Vanda boost sales of Fanapt to $84 million over the first nine months of 2025, a 24% year-over-year increase.
Vanda also sells sleep disorder drug Hetlioz, which reached the market in 2014, and relapsing multiple sclerosis treatment Ponvory, which was approved in 2021. The FDA is scheduled on Jan. 7 to decide on Vanda’s Hetlioz as a treatment for jet lag disorder.
As for tradipitant, Vanda hasn’t surrendered its effort to get it across the FDA finish line for gastroparesis. Vanda also is studying tradipitant to prevent nausea and vomiting induced by GLP-1 obesity drugs.