Marstacimab-hncq (Hympavzi – Pfizer), a tissue factor pathway inhibitor (TFPI) antagonist, has been approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ≥12 years old who have hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. It is the first hemophilia treatment to be approved in the US that targets an inhibitor of coagulation and the first to become available in prefilled pens and syringes for subcutaneous administration.

Concizumab (Alhemo – Novo Nordisk), a subcutaneously injected tissue factor pathway inhibitor (TFPI) antagonist, has been approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ≥12 years old who have hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors. It is the second TFPI antagonist to be approved in the US for treatment of hemophilia A or B; the TFPI antagonist marstacimab (Hympavzi) is approved for use in patients without inhibitors.

The FDA has approved vutrisiran (Amvuttra – Alnylam), a subcutaneously injected small interfering RNA (siRNA), to reduce cardiovascular hospitalizations, urgent heart failure visits, and cardiovascular death in adults with wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM). Vutrisiran is the first siRNA to be approved in the US for this indication; it was approved earlier for treatment of polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

The FDA has approved Brekiya (Amneal), the first dihydroergotamine (DHE) autoinjector product. DHE has been available for IV, IM, SC and intranasal administration (Trudhesa, and others). Like other parenteral DHE products, Brekiya is indicated for the acute treatment of migraine, with or without aura, and cluster headache in adults. It is not recommended for treatment of hemiplegic or basilar migraine. The FDA also recently approved the first intranasal powder formulation of DHE (Atzumi) for acute migraine treatment; it will be reviewed in a future issue.

The FDA has approved imlunestrant (Inluriyo – Lilly), an oral estrogen receptor antagonist, for treatment of estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer in adults who had disease progression following at least one prior line of endocrine therapy. Imlunestrant is the second oral selective estrogen receptor degrader (SERD) to be approved in the US for treatment of breast cancer; elacestrant (Orserdu) was approved in 2023.

Obinutuzumab (Gazyva – Genentech), a CD20-directed monoclonal antibody, has been approved by the FDA for treatment of adults with active lupus nephritis who are receiving standard treatment. It was previously approved for treatment of chronic lymphocytic leukemia and follicular lymphoma. Obinutuzumab is the third drug to be approved in the US for treatment of lupus nephritis; the oral calcineurin inhibitor voclosporin (Lupkynis) and the parenteral B-lymphocyte stimulator (BlyS)-specific inhibitor belimumab (Benlysta) were approved earlier.

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 ensitrelvir (Xocova – Shionogi), a SARS-CoV-2 main protease inhibitor, for postexposure prophylaxis of COVID-19 in patients ≥12 years old who had contact with an infected individual. Ensitrelvir is the first oral antiviral drug to be approved in the US for this indication. The parenteral monoclonal antibody combinations bamlanivimab and etesevimab and casirivimab and imdevimab (REGEN-COV) are no longer authorized for postexposure prophylaxis of COVID-19 in the US.

Vepdegestrant (Veppanu – Rigel), an oral heterobifunctional protein degrader, has been approved by the FDA for treatment of estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, estrogen receptor 1 (ESR1)-mutated advanced or metastatic breast cancer in adults who had disease progression following ≥1 prior line of endocrine therapy. It is the first proteolysis targeting chimera (PROTAC), a type of heterobifunctional protein degrader, to be approved in the US. The oral selective estrogen receptor degraders (SERDs) elacestrant (Orserdu) and...

The FDA has licensed mFLUSIVA (Moderna), the first mRNA-based influenza vaccine in the US, for prevention of influenza in adults ≥50 years old. mFLUSIVA will be available for the 2026-2027 influenza season. Three mRNA COVID-19 vaccines (Comirnaty, Spikevax, and mNEXSPIKE) and one mRNA RSV vaccine (mRESVIA) are also licensed in the US.