Temsirolimus (Torisel - Wyeth), an mTOR (mammalian target of rapamycin) kinase inhibitor that is metabolized to sirolimus (rapamycin), has been approved by the FDA for intravenous treatment of advanced renal cell carcinoma.

Sebetralstat (Ekterly – Kalvista), an oral plasma kallikrein inhibitor, has been approved by the FDA for treatment of acute hereditary angioedema (HAE) attacks in patients ≥12 years old. It is the first oral drug to be approved in the US for this indication. The intravenous C1 esterase inhibitors (C1INHs) Berinert and Ruconest, the subcutaneous plasma kallikrein inhibitor ecallantide (Kalbitor), and the subcutaneous bradykinin B2 receptor antagonist icatibant (Firazyr) have been available for years for on-demand treatment of HAE attacks; all of these drugs except ecallantide can...

Linezolid, the first of a new class of antibiotics, the oxazolidinones, has been marketed for treatment of infection due to vancomycin-resistant Enterococcus faecium, nosocomial and community-acquired pueumonia due to Staphylococcus aureus or penicillin-sucsceptible Streptococcus pneumoniae, and skin and skin structure infections, including those due to methicillin-resistant S. aureus (MRSA).

The injectable interleukin (IL)-23 antagonist guselkumab (Tremfya – Janssen Biotech) has now been approved by the FDA for treatment of moderately to severely active Crohn's disease (CD) in adults; it was approved earlier for treatment of ulcerative colitis (UC), plaque psoriasis, and psoriatic arthritis. The IL-23 antagonists risankizumab (Skyrizi) and mirikizumab (Omvoh) and the IL-12/23 antagonist ustekinumab (Stelara, and biosimilars) are also approved for treatment of CD.

Yutrepia (Liquidia), a dry powder inhaler formulation of the prostacyclin analog treprostinil, has been approved by the FDA to improve exercise ability in adults with pulmonary arterial hypertension (PAH; WHO Group 1) or pulmonary hypertension associated with interstitial lung disease (PH-ILD; WHO Group 3). Tyvaso DPI, another treprostinil dry powder inhaler, was approved earlier for the same indication.

The FDA has approved mavacamten (Camzyos – MyoKardia/BMS), a modulator of cardiac myosin, to improve functional capacity and symptoms in adults with New York Heart Association (NYHA) class II or III obstructive hypertrophic cardiomyopathy (HCM) who have a baseline left ventricular ejection fraction (LVEF) ≥55%. Mavacamten is the first drug in its class to become available in the US.

The potassium-competitive acid blocker vonoprazan (Voquezna – Phathom), which was approved earlier for treatment of erosive esophagitis, has now been approved by the FDA for relief of heartburn associated with nonerosive gastroesophageal reflux disease (GERD) in adults. Vonoprazan is also available copackaged with amoxicillin (Voquezna Dual Pak) and with amoxicillin and clarithromycin (Voquezna Triple Pak) for treatment of Helicobacter pylori infection.

Caspofungin acetate(Cancidas) is the first of a new class of antifungals, the echinocandins. It has been approved by the FDA for treatment of invasive aspergillosis in patients who fail to respond to or are unable to tolerate other antifungal drugs such as amphotericin B and itraconazole.

Ziftomenib (Komzifti – Kura), an oral menin inhibitor, has been approved by the FDA for treatment of adults with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options. It is the second oral menin inhibitor to be approved in the US for this indication; revumenib (Revuforj) was approved in October 2025 for use in patients ≥1 year old.

The FDA has approved Zunveyl (Alpha Cognition), a delayed-release formulation of the prodrug benzgalantamine, for treatment of mild to moderate dementia of Alzheimer's disease (AD). Immediate- and extended-release formulations of the acetylcholinesterase inhibitor galantamine have been available in the US for years for treatment of AD.