A reader of our article on the 2024-2025 formulations of the mRNA COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) asked us to provide more information on the data that supported their licensure.

Lazertinib (Lazcluze – Janssen Biotech), an oral kinase inhibitor, has been approved by the FDA for use in combination with the EGFR-MET bispecific antibody amivantamab (Rybrevant) for first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations. This is the first approval for Lazcluze; amivantamab was previously approved for use alone and in combination with carboplatin and pemetrexed for treatment of NSCLC with EGFR exon 20 insertion...

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 FDA has approved diazoxide choline extended-release tablets (Vykat XR – Soleno Therapeutics) for treatment of hyperphagia in patients ≥4 years old with Prader-Willi syndrome. Diazoxide choline is the first drug to be approved in the US for this indication. Diazoxide oral suspension (Proglycem) has been available for years for management of symptomatic hypoglycemia.

The FDA has licensed two new pneumococcal conjugate vaccines (PCVs) for prevention of invasive pneumococcal disease in adults: Prevnar 20 (PCV20; Pfizer), which contains antigens from 20 serotypes of pneumococcus, and Vaxneuvance (PCV15; Merck), which contains antigens from 15 serotypes. Two other pneumococcal vaccines are available in the US: Prevnar 13 (PCV13; Pfizer), a 13-valent conjugate vaccine licensed for use in persons ≥6 weeks old, and Pneumovax 23 (PPSV23; Merck), a 23-valent pneumococcal polysaccharide vaccine licensed for use in persons ≥2 years...

The FDA has approved Alyftrek (Vertex), an oral fixed-dose combination of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators vanzacaftor, tezacaftor, and deutivacaftor, for once-daily treatment of cystic fibrosis (CF) in patients ≥6 years old who have at least one F508del mutation or another responsive mutation in the CFTR gene. This is the first approval for vanzacaftor and for deutivacaftor, a deuterated form of ivacaftor. Trikafta, a twice-daily oral fixed-dose combination of elexacaftor, tezacaftor, and ivacaftor, is FDA-approved for the same indication...

The B-lymphocyte stimulator (BLyS)-specific inhibitor belimumab (Benlysta – GSK), which was approved earlier for treatment of active, autoantibody-positive, nonrenal, systemic lupus erythematosus (SLE), has now been approved for use in addition to standard therapy for treatment of active lupus nephritis in adults. Belimumab is the first drug to be approved in the US for treatment of both SLE and lupus nephritis.

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.

The FDA has approved Twyneo (Galderma), a cream containing the retinoid tretinoin and the oxidizing agent benzoyl peroxide, for once-daily topical treatment of acne in patients ≥9 years old.