The FDA has approved abemaciclib (Verzenio – Lilly), an oral cyclin-dependent kinase (CDK) 4/6 inhibitor, for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer. Abemaciclib is the third CDK 4/6 inhibitor to be approved in the US for this indication.

The FDA has approved benralizumab (Fasenra – AstraZeneca), a humanized monoclonal antibody selective for the interleukin-5 (IL-5) receptor, for add-on maintenance treatment of severe asthma in patients ≥12 years old with an eosinophilic phenotype. Benralizumab is the third anti-IL-5 antibody to be approved for treatment of severe eosinophilic asthma; mepolizumab (Nucala) and reslizumab (Cinqair), which target IL-5 itself, were approved earlier.

The FDA has approved voretigene neparvovec-rzyl intraocular suspension (Luxturna – Spark), an adeno-associated virus vector-based gene therapy, for treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy in patients who have viable retinal cells. It is the first gene replacement therapy approved in the US that targets a disease caused by mutations in a specific gene and the first pharmacologic treatment to be approved for this disorder.

The FDA has approved brigatinib (Alunbrig – Takeda), an oral tyrosine kinase inhibitor, for treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib (Xalkori). Translocations of the ALK gene are found in about 5% of lung cancers; they occur predominantly in nonsmokers with adenocarcinoma. Brigatinib is the third tyrosine kinase inhibitor to be approved for this indication; ceritinib (Zykadia) and alectinib (Alecensa) were approved earlier, and subsequently were...

The FDA has approved inotuzumab ozogamicin (Besponsa – Pfizer), a humanized anti-CD22 monoclonal antibody conjugated to the cytotoxic antibiotic calicheamicin, for treatment of relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL) in adults. It is the first CD22-directed antibody-drug conjugate to be approved in the US.

The FDA has approved an extended-release oral suspension formulation of amphetamine (Adzenys ER – Neos) for treatment of attention deficit hyperactivity disorder (ADHD) in patients ≥6 years old. The same extended-release amphetamine product is available as an orally disintegrating tablet and is marketed as Adzenys XR-ODT. Another extended-release oral suspension formulation of amphetamine, Dyanavel XR, was approved earlier.

The FDA has approved elagolix (Orilissa – AbbVie), an oral gonadotropin-releasing hormone (GnRH) antagonist, for management of moderate to severe pain associated with endometriosis.

The FDA has approved the fixed-dose combination of tezacaftor and ivacaftor (Symdeko – Vertex) for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who are homozygous for the F508del (also called Phe508del or ΔF508) mutation or have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the combination. About 50% of CF patients in the US are homozygous for the F508del mutation. This is the first approved indication for tezacaftor. Ivacaftor is also available in combination with lumacaftor...

The FDA has approved a sublingual tablet (Nocdurna – Ferring) and a nasal spray (Noctiva – Avadel) formulation of desmopressin acetate for treatment of nocturia due to nocturnal polyuria in adults who wake up ≥2 times per night to void. Nocdurna and Noctiva are the first two products to be approved in the US for this indication. Higher-strength nasal spray and oral tablet formulations of desmopressin (DDAVP, and others) have been available for years for treatment of diabetes insipidus and primary nocturnal enuresis (bedwetting in children). In 2007, the indication for primary...

The FDA has approved solriamfetol (Sunosi – Jazz), a dopamine and norepinephrine reuptake inhibitor (DNRI), to improve wakefulness in adults with excessive daytime sleepiness (EDS) associated with narcolepsy or obstructive sleep apnea (OSA). Pitolisant (Wakix), an H3-receptor antagonist/inverse agonist recently approved by the FDA for treatment of excessive daytime sleepiness in patients with narcolepsy, will be reviewed in a future issue.