The FDA has approved the nitrofuran antiprotozoal drug nifurtimox (Lampit – Bayer) for treatment of Chagas disease (American trypanosomiasis) in children who weigh ≥2.5 kg. Nifurtimox was previously available in the US only through a CDC-sponsored Investigational New Drug (IND) program.

Mitapivat (Aqvesme – Agios), an oral pyruvate kinase activator, has been approved by the FDA for treatment of anemia in adults with alpha or beta thalassemia. It is the first product to be approved in the US for treatment of alpha thalassemia and the first oral option for beta thalassemia. Mitapivat was approved in 2022 as Pyrukind for treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

The oral second-generation antipsychotic drug lumateperone (Caplyta – Intra-Cellular Therapies), which was approved by the FDA in 2020 for treatment of schizophrenia, is now approved for use as monotherapy or as an adjunct to lithium or valproate for treatment of depressive episodes associated with bipolar I or II disorder in adults.

The FDA has approved a 12.45% gel formulation of the anticholinergic drug sofpironium (Sofdra – Botanix) for treatment of primary axillary hyperhidrosis (excessive underarm sweating) in patients ≥9 years old.

Breztri Aerosphere (AstraZeneca), a three-drug inhaler containing the inhaled corticosteroid (ICS) budesonide, the long-acting antimuscarinic agent (LAMA) glycopyrrolate, and the long-acting beta2-agonist (LABA) formoterol fumarate, has been approved by the FDA for maintenance treatment of asthma in patients ≥12 years old. It was approved in 2020 for treatment of chronic obstructive pulmonary disease (COPD). Breztri Aerosphere is the second ICS/LAMA/LABA combination to become available for treatment of asthma. Trelegy Ellipta, which contains the ICS fluticasone, the...

Palopegteriparatide (Yorvipath – Ascendis), a parathyroid hormone (PTH) 1-34 analog, has been approved by the FDA for treatment of hypoparathyroidism in adults. The parathyroid hormone analog teriparatide (Forteo, and others), which is approved for treatment of postmenopausal osteoporosis, has been used off-label for treatment of hypoparathyroidism.

Atogepant (Qulipta – Abbvie), an oral small-molecule calcitonin gene-related peptide (CGRP) receptor antagonist ("gepant"), has been approved by the FDA for prevention of episodic migraine in adults. It is the second oral CGRP receptor antagonist to be approved in the US for this indication; the first was rimegepant (Nurtec ODT), which is also approved for acute treatment of migraine. Parenteral CGRP monoclonal antibodies are approved for prevention of migraine (see Table 3).

The FDA has approved orforglipron (Foundayo – Lilly), an oral small-molecule, nonpeptide glucagon-like peptide-1 (GLP-1) receptor agonist, to reduce excess body weight and maintain weight reduction long term in adults with obesity, or with overweight and at least one weight-related comorbidity. Orforglipron is the second oral GLP-1 receptor agonist to be approved in the US for this indication; an oral formulation of the peptide semaglutide (Wegovy) was approved in 2025.

The FDA has approved baxdrostat (Baxfendy – AstraZeneca), an oral aldosterone synthase inhibitor, for add-on treatment of hypertension in adults whose blood pressure is not adequately controlled on other drugs. Baxdrostat is the first aldosterone synthase inhibitor to become available in the US.

The FDA has approved an oral extended-release formulation of gepirone (Exxua – Fabre-Kramer), a selective 5-HT_1A receptor agonist, for treatment of major depressive disorder (MDD) in adults. Gepirone was originally developed decades ago for treatment of anxiety and depression, but it was not approved by the FDA for such use. Gepirone is structurally related to buspirone, which is FDA-approved for treatment of anxiety.