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.

Itraconazole (Sporanox - Janssen), an oral antifungal triazole that has been available in the USA since 1992 for treatment of deep fungal infections (Medical Letter, 35:7, 1993), is now being marketed for treatment of dermatophyte infections of the toenails, with or without fingernail involvement.

The FDA has approved efinaconazole 10% solution (Jublia – Valeant) for topical treatment of toenail onychomycosis due to Trichophyton rubrum and/or Trichophyton mentagrophytes. It is the first topical triazole antifungal drug to be approved for this indication. A topical oxaborole antifungal solution, tavaborole 5% (Kerydin – Anacor), also recently approved by the FDA for the same indication will be reviewed in a future issue.

The FDA has approved mepolizumab (Nucala - GSK), a subcutaneously injected humanized interleukin-5 (IL-5) antagonist monoclonal antibody, for maintenance treatment of severe asthma in patients ≥12 years old who have an eosinophilic phenotype.

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.

Eflapegrastim-xnst ((Rolvedon) [Spectrum]; previously called Rolontis), a granulocyte colony-stimulating factor (G-CSF) conjugated to an Fc fragment of human IgG4, has been approved by the FDA to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive chemotherapy that can cause clinically significant febrile neutropenia.

Fruquintinib (Fruzaqla – Takeda), an oral kinase inhibitor, has been approved by the FDA for treatment of adults with metastatic colorectal cancer (mCRC) who received prior fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, anti-VEGF therapy, and, in patients with RAS wild-type mutations, anti-EGFR therapy. The drug can be used in patients with mCRC regardless of biomarker status. Fruquintinib is the first drug to become available in the US for treatment of mCRC that targets 3 VEGF receptor kinases.