Tofersen (Qalsody – Biogen), an intrathecally administered antisense oligonucleotide, has received accelerated approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. About 2% of patients with ALS have mutations in the SOD1 gene. Tofersen is the first drug to be approved in the US that targets a genetic cause of ALS. Accelerated approval of the drug was based on the surrogate endpoint of a reduction in plasma neurofilament light chain, which is considered likely to predict clinical...

The oral polymerase acidic endonuclease inhibitor baloxavir marboxil (Xofluza — Genentech) is now FDAapproved for post-exposure prophylaxis of influenza in patients ≥12 years old. Baloxavir was approved for treatment of acute uncomplicated influenza in patients ≥12 years old in 2018.1 Two neuraminidase inhibitors are FDA-approved for prophylaxis of influenza: oseltamivir (Tamiflu, and generics) in patients ≥1 year old, and zanamivir (Relenza) in patients ≥5 years old.2 Unlike oseltamivir and zanamivir, baloxavir is not FDA-approved for pre-exposure prophylaxis of...

A transdermal formulation of the second-generation (atypical) antipsychotic asenapine (Secuado – Noven) has been approved by the FDA for once-daily treatment of schizophrenia in adults. Asenapine is the first antipsychotic to become available in a transdermal formulation in the US. A twice-daily sublingual tablet formulation of asenapine (Saphris) has been available since 2009.

The FDA has approved osimertinib (Tagrisso – AstraZeneca), an oral kinase inhibitor, for adjuvant treatment of non-small cell lung cancer (NSCLC) after tumor resection in adults who have epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations. Osimertinib is the first targeted therapy to be approved in the US for this indication. The drug was previously approved for first-line treatment of adults with NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations and for treatment of EGFR T790M mutation-positive NSCLC in adults whose disease progressed...

The FDA has approved mavacamten (Camzyos – MyoKardia/BMS), a modulator of cardiac myosin, to improve functional capacity and symptoms in adults with New York Heart Association (NYHA) class II or III obstructive hypertrophic cardiomyopathy (HCM) who have a baseline left ventricular ejection fraction (LVEF) ≥55%. Mavacamten is the first drug in its class to become available in the US.

The IV antiviral drug remdesivir (Veklury – Gilead) has been available for treatment of COVID-19 in hospitalized patients since 2020. Now, the FDA has approved remdesivir for treatment of mild to moderate COVID-19 in outpatients ≥12 years old who weigh ≥40 kg and are at high risk for progression to severe disease, including hospitalization or death; they also issued an Emergency Use Authorization (EUA) allowing its use in any other high-risk outpatient who weighs ≥3.5 kg.

The FDA has approved oral and injectable formulations of the HIV-1 capsid inhibitor lenacapavir (Sunlenca – Gilead) for use with other antiretroviral drugs to treat multidrug-resistant HIV-1 infection (MDR-HIV) in heavily treatment-experienced adults whose current regimen is failing. Lenacapavir is the third drug to be approved exclusively for treatment of MDR-HIV; the intravenously administered CD4-directed antibody ibalizumab-uiyk (Trogarzo) and the orally administered HIV-1 gp120-directed attachment inhibitor fostemsavir (Rukobia) were approved earlier.

Casimersen (Amondys 45 – Sarepta), an IV antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 45 skipping (DMD-45), which occur in ~8% of DMD cases. Casimersen is the first drug to be approved for this indication and the fifth to be approved for treatment of DMD; the IV antisense oligonucleotides eteplirsen (Exondys 51), golodirsen (Vyondys 53), and viltolarsen (Viltepso) and the oral corticosteroid deflazacort (Emflaza) were...

The FDA has approved a higher-dose injectable formulation of the opioid antagonist naloxone (Zimhi – Adamis) for emergency treatment of opioid overdose. A single IM or SC injection of the new formulation delivers 5 mg of naloxone; injectable formulations that deliver 0.4 mg or 2 mg of the drug have been available for years. Naloxone is also available in intranasal formulations for the same indication (see Table 1).

Efgartigimod alfa-fcab (Vyvgart – Argenx), a first-in-class neonatal Fc receptor (FcRn) antagonist, has been approved by the FDA for IV treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. The IV complement inhibitor eculizumab (Soliris) was approved for the same indication in 2017.