The Bruton's tyrosine kinase (BTK) inhibitor zanubrutinib (Brukinsa), which was previously approved by the FDA for treatment of mantle cell lymphoma, Waldenström's macroglobulinemia, and relapsed or refractory marginal zone lymphoma, has now been approved for treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adults. The BTK inhibitors ibrutinib (Imbruvica) and acalabrutinib (Calquence) were approved earlier for treatment of CLL and SLL.

Atezolizumab (Tecentriq – Genentech), an immune checkpoint inhibitor, has been approved by the FDA for treatment of unresectable or metastatic alveolar soft part sarcoma (ASPS) in patients ≥2 years old. It was previously approved for treatment of non-small cell lung cancer, small cell lung cancer, hepatocellular cancer, and melanoma (see Table 1). Atezolizumab is the first drug to be approved in the US for treatment of ASPS. ASPS is a rare disorder that affects mostly adolescents and young adults; <1% of soft tissue sarcomas are ASPS.

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.

Isatuximab-irfc (Sarclisa – Sanofi), a CD38-directed cytolytic antibody, has been approved by the FDA for treatment of newly diagnosed multiple myeloma in adults who are not eligible for autologous stem cell transplantation (ASCT). The drug was approved earlier for treatment of relapsed or treatment-refractory multiple myeloma.

Vimseltinib (Romvimza – Deciphera), an oral kinase inhibitor, has been approved by the FDA for treatment of symptomatic tenosynovial giant cell tumor in adults for whom surgical resection would cause worsening functional limitation or severe morbidity. The kinase inhibitor pexidartinib (Turalio) was approved for a similar indication in 2019.

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Relyvrio, the fixed-dose combination of sodium phenyl-butyrate and taurursodiol that received accelerated approval for treatment of amyotrophic lateral sclerosis (ALS) in 2022 based on a phase 2 trial showing that it slowed functional decline, has voluntarily been withdrawn from the market. According to the manufacturer (Amylyx), the decision to withdraw Relyvrio was based on the results of a phase 3, 48-week trial (PHOENIX). The change from baseline on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, which measures fine motor, gross motor, bulbar, and respiratory...

The FDA has approved anifrolumab-fnia (Saphnelo – AstraZeneca), a type I interferon receptor antagonist, for IV treatment of adults with moderate to severe systemic lupus erythematosus (SLE) who are receiving standard treatment. Anifrolumab has not been studied in patients with severe active lupus nephritis or severe active CNS lupus. It is the first type I interferon receptor antagonist to become available in the US.

Opdualag (BMS), a fixed-dose combination of two immune checkpoint inhibitors — nivolumab (Opdivo), a programmed death receptor-1 (PD-1) inhibitor, and relatlimab-rmbw, a lymphocyte-activation gene-3 (LAG-3) blocking antibody — has been approved by the FDA for treatment of unresectable or metastatic melanoma in patients ≥12 years old. Relatlimab, which is only available in combination with nivolumab, is the first LAG-3 blocking antibody to become available in the US. Immune checkpoint inhibitors, including the anti-CTLA-4 antibody ipilimumab (Yervoy) and the PD-1 inhibitors...

The FDA has approved Rebyota (Ferring), a rectally-administered, live fecal microbiota suspension, for prevention of additional recurrences of Clostridioides difficile infection (CDI) following antibiotic treatment for a recurrent episode of CDI in adults. Rebyota is the first microbiome-based treatment to be approved for this indication. It is not approved for initial treatment of CDI. Fecal microbiota transplantation (FMT) has been used in patients with multiple CDI recurrences.