The Bruton's tyrosine kinase (BTK) inhibitor pirtobrutinib (Jaypirca – Lilly) has received accelerated approval from the FDA for treatment of relapsed or refractory mantle cell lymphoma (MCL) in adults who received ≥2 prior lines of systemic therapy, including a BTK inhibitor. Accelerated approval was based on the response rate. The BTK inhibitors ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa) are also approved for treatment of MCL.

The oral cyclin-dependent kinase (CDK) 4/6 inhibitor ribociclib (Kisqali – Novartis), which was approved by the FDA in 2017 for use with an aromatase inhibitor or fulvestrant for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, has now been approved for use with an aromatase inhibitor for adjuvant treatment of HR-positive, HER2-negative stage II or III early breast cancer at high risk of recurrence. Kisqali can be used in patients with node-positive or node-negative disease. The CDK 4/6...

Revumenib (Revuforj – Syndax), an oral menin inhibitor, has been approved by the FDA for treatment of relapsed or refractory acute leukemia in patients ≥1 year old with a lysine methyltransferase 2A gene (KMT2A) translocation. It is the first menin inhibitor to be approved in the US.

Mirdametinib (Gomekli – SpringWorks Therapeutics), an oral kinase inhibitor, has been approved by the FDA for treatment of neurofibromatosis type 1 (NF1) in patients ≥2 years old who have symptomatic plexiform neurofibromas not amenable to complete resection. Mirdametinib is the second drug to be approved for this indication in the US and the first to be approved for use in adults. The oral kinase inhibitor selumetinib (Koselugo) was approved in 2020 for use in patients 2-17 years old.

The investigational neutralizing IgG1 monoclonal antibody bamlanivimab (LY-CoV555; Lilly) has been granted an FDA Emergency Use Authorization (EUA) for treatment of recently diagnosed mild to moderate COVID-19 in patients who are ≥12 years old, weigh at least 40 kg, and are at high risk for progressing to severe disease and/or hospitalization (see Table 1).

Odevixibat (Bylvay – Albireo), an oral ileal bile acid transporter (IBAT) inhibitor, has been approved by the FDA for treatment of pruritus in patients ≥3 months old with progressive familial intrahepatic cholestasis (PFIC). It is the first drug to be approved in the US for this indication.

Tovorafenib (Ojemda – Day One), a type II RAF kinase inhibitor, has received accelerated approval from the FDA for treatment of patients ≥6 months old with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement or a BRAF V600 mutation. Tovorafenib is the first systemic treatment to be approved in the US for pediatric low-grade gliomas with BRAF fusions. Accelerated approval of tovorafenib was based on response rates and duration of response.

The investigational monoclonal antibodies casirivimab and imdevimab (REGEN-COV – Regeneron) have been available in the US under an Emergency Use Authorization (EUA) since late 2020 for use together to treat mild to moderate COVID-19 in persons ≥12 years old who weigh ≥40 kg and are at high risk of progression to severe disease or hospitalization. The FDA has now expanded this EUA to allow use of the antibodies together for post-exposure prophylaxis of COVID-19 in such persons, if they are not fully vaccinated against COVID-19 or are unlikely to have an adequate immune response...

On December 22, 2021, the FDA issued an Emergency Use Authorization (EUA) for the investigational antiviral drug nirmatrelvir copackaged with the HIV-1 protease inhibitor ritonavir (Paxlovid – Pfizer) for oral treatment of mild to moderate COVID-19 in outpatients ≥12 years old who weigh at least 40 kg and are at high risk of progressing to severe disease, including hospitalization or death. Paxlovid was the first oral antiviral drug to be authorized in the US for treatment of COVID-19; Merck's oral antiviral drug molnupiravir was granted an EUA for treatment of COVID-19 on...

Adagrasib (Krazati – Mirati Therapeutics), a RAS GTPase family inhibitor, has received accelerated approval from the FDA for oral treatment of KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults who received at least one prior systemic therapy. Accelerated approval was based on the objective response rate and duration of response.