Afamitresgene autoleucel (Tecelra – Adaptimmune), a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T-cell immunotherapy, has received accelerated approval from the FDA for one-time treatment of adults with unresectable or metastatic synovial sarcoma who received prior chemotherapy and are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen. It is the first gene therapy to be approved in the US for treatment of synovial sarcoma. Accelerated approval of the immunotherapy was based on the...

Zanidatamab-hrii (Ziihera – Jazz), a bispecific HER2-directed antibody, has received accelerated approval from the FDA for treatment of unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer in patients who received prior therapy. Biliary tract cancer includes gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma. Zanidatamab is the first dual HER2-targeted therapy to be approved in the US for this indication. Accelerated approval was based on the overall response rate and duration of response.

Remestemcel-L (Ryoncil – Mesoblast), an allogeneic bone marrow-derived mesenchymal stromal cell therapy, has been approved by the FDA for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in patients ≥2 months old. Steroid-refractory GVHD is associated with a poor prognosis, organ damage, and death, particularly in pediatric patients. Remestemcel-L is the first cellular therapy to be approved in the US for this indication and the only treatment of any kind to be approved for GVHD in children <12 years old.

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.

The FDA has expanded its Emergency Use Authorizations (EUAs) for the mRNA COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) to allow for their use in children as young as 6 months old. The Pfizer vaccine was previously authorized for use in persons ≥5 years old, and the Moderna vaccine was authorized for use in adults ≥18 years old.

The injectable interleukin (IL)-23 antagonist risankizumab-rzaa (Skyrizi – Abbvie) has been approved by the FDA for treatment of moderately to severely active Crohn's disease (CD) in adults. Risankizumab was approved earlier for treatment of plaque psoriasis and psoriatic arthritis.

The FDA has approved elacestrant (Orserdu – Stemline), 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 postmenopausal women or men who had disease progression following endocrine therapy. Elacestrant is the first oral selective estrogen receptor degrader (SERD) to be approved for treatment of breast cancer; the injectable SERD fulvestrant (Faslodex, and generics) was approved more than 20 years...

View the Comparison Table: Some Drugs for Postmenopausal Osteoporosis

The FDA has approved pilocarpine 0.4% preservativefree ophthalmic solution (Qlosi – Orasis) for treatment of presbyopia in adults. Pilocarpine 1.25% ophthalmic solution (Vuity) was approved in 2021 for the same indication. Pilocarpine 1%, 2%, and 4% ophthalmic solutions (Isopto Carpine, and others) have been available for years for treatment of glaucoma, but local (e.g., blurry vision and instillation-site pain) and systemic (e.g., sweating and GI disturbances) adverse effects have limited their use.

The FDA has approved the complement factor B inhibitor iptacopan (Fabhalta – Novartis) for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults. Iptacopan is the first oral drug to be approved in the US for this indication. Three parenterally administered drugs, the complement C5 inhibitors eculizumab (Soliris) and ravulizumab (Ultomiris) and the complement C3 inhibitor pegcetacoplan (Empaveli), are also approved for treatment of PNH.