Metreleptin (Myalept – Amylin), a recombinant leptin analog produced in E. coli, has been approved by the FDA to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. It has not been approved to date for the treatment of partial lipodystrophies, including those associated with the use of protease inhibitors in patients with HIV. Metreleptin is approved in Japan for the treatment of any lipodystrophy disorder.

The FDA has approved the interleukin-6 (IL-6) receptor antagonist tocilizumab (Actemra – Genentech) for subcutaneous (SC) treatment of giant cell arteritis in adults. It is the first drug to be approved in the US for this indication. Tocilizumab is also approved for treatment of rheumatoid arthritis, polyarticular or systemic juvenile idiopathic arthritis, and cytokine release syndrome.

The FDA has approved lanadelumab-flyo (Takhzyro – Takeda), a subcutaneously-administered plasma kallikrein inhibitor, for prevention of hereditary angioedema (HAE) attacks in adults and children ≥12 years old. Lanadelumab is the third drug to be approved for this indication; the human plasmaderived C1 esterase inhibitors (C1INHs) Cinryze, which is administered IV, and Haegarda, which is given SC, have been available for years for HAE prophylaxis in patients ≥6 years old.

The FDA has approved Acuvue Theravision with Ketotifen (Johnson & Johnson Vision Care), a daily-wear, disposable, vision-correcting soft contact lens that releases the H₁-antihistamine ketotifen, for prevention of ocular itch due to allergic conjunctivitis in contact lens users. This is the first approval for a drug-eluting contact lens. Ketotifen eye drops (Alaway, Zaditor, and others) are available over the counter.

Epcoritamab-bysp (Epkinly – Genmab), a bispecific CD20-directed CD3 T-cell engager, has received accelerated approval from the FDA for subcutaneous treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after ≥2 lines of systemic therapy. Epcoritamab is the second T-cell-engaging bispecific antibody to become available in the US for treatment of non-Hodgkin's lymphoma; mosunetuzumab-axgb (Lunsumio) was recently approved for relapsed or refractory...

Talquetamab-tgvs (Talvey – Janssen), a bispecific GPRC5D-directed T-cell engager, has been granted accelerated approval by the FDA for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Accelerated approval was based on the response rate and durability of response. Talquetamab is the first bispecific GPRC5D-directed T-cell engager to be approved in the US

Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.

Natalizumab, a recombinant humanized monoclonal antibody, has received accelerated approval from the FDA for intravenous treatment of relapsing forms of multiple sclerosis (MS). The beta interferons and glatiramer acetate are widely used for treatment of MS; they generally reduce the number of relapses by about 30% compared to placebo, and have been shown to be safe and effective for periods ranging from 4 to 10 years.

The FDA has approved the marketing of Resperate (InterCure), a device that helps patients breathe slowly, as an adjunct for treatment of hypertension. The FDA does not require proof of effectiveness for approval of devices with minimal potential for harm, such as this one.

The FDA has approved Trikafta (Vertex), a fixed-dose combination of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators elexacaftor, tezacaftor, and ivacaftor, for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who have at least one Phe508del mutation in the CFTR gene. About 90% of patients with CF have at least one copy of the Phe508del (also called F508del) mutation. This is the first approval for elexacaftor. Ivacaftor is available alone (Kalydeco) and in 2-drug combinations with tezacaftor (Symdeco) and lumacaftor...