Ferric carboxymaltose (Injectafer – American Regent) is now FDA-approved for IV treatment of iron deficiency in adults with New York Heart Association (NYHA) class II/III heart failure to improve exercise capacity. Iron deficiency is present in approximately 30% of patients with heart failure; it has been associated with reduced quality of life and increased hospitalization and mortality. Injectafer is the first IV iron preparation to be approved for this indication. It was previously approved for treatment of iron deficiency anemia in children and adults.

The FDA has approved ganaxolone (Ztalmy – Marinus) for oral treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder in patients ≥2 years old. It is the first drug to be approved in the US for this indication.

The FDA has approved the complement factor B inhibitor iptacopan (Fabhalta – Novartis) for treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults. Iptacopan is the first oral drug to be approved in the US for this indication. Three parenterally administered drugs, the complement C5 inhibitors eculizumab (Soliris) and ravulizumab (Ultomiris) and the complement C3 inhibitor pegcetacoplan (Empaveli), are also approved for treatment of PNH.

The injectable interleukin (IL)-23 antagonist guselkumab (Tremfya – Janssen Biotech) has now been approved by the FDA for treatment of moderately to severely active Crohn's disease (CD) in adults; it was approved earlier for treatment of ulcerative colitis (UC), plaque psoriasis, and psoriatic arthritis. The IL-23 antagonists risankizumab (Skyrizi) and mirikizumab (Omvoh) and the IL-12/23 antagonist ustekinumab (Stelara, and biosimilars) are also approved for treatment of CD.

The erythropoiesis-stimulating agents (ESAs) epoetin alfa (Epogen, Procrit) and darbepoetin alfa (Aranesp) are widely used for treatment of anemia and to reduce the need for red blood cell transfusions. Based on the results of recent clinical trials indicating an increased risk of serious adverse events and death associated with ESAs, particularly when used to achieve a hemoglobin concentration ≥12 g/dL, the FDA has revised the prescribing information for these drugs to include a black box warning.

Desmopressin acetate (DDAVP -Rorer), a synthetic analog of the natural human antidiuretic hormone arginine vasopressin, was recently approved by the US Food and Drug Administration for treatment of primary nocturnal enuresis. DDAVP Nasal Spray, which is similar to an intranasal solution that has been available in the USA for treatment of central diabetes insipidus (Medical Letter, 20:26, 1978), will be used for the new indication.

Interferon β-1a (Avonex - Biogen), a recombinant form of human interferon β, has been approved by the US Food and Drug Administration (FDA) for treatment of relapsing forms of multiple sclerosis (MS). Interferon β-1a (Betaseron - Berlex), a recombinant analog of interferon β that differs from interferon β-1a in having a serine substituted for a cysteine and in not being glycosylated, was previously approved for this indication (Medical Letter, 35:61, 1993; BW van Oosten et al, Drugs, 49:200, 1995).

Myocardial perfusion imaging and stress echocardiography in patients who cannot exercise may require use of drugs. Arbutamine (GenESA - Gensia Automedics), a potent synthetic sympathomimetic, has recently been approved by the FDA for use with a computerized drug-delivery system in pharmacologic stress testing.

Temozolomide has received accelerated approval from the FDA for oral treatment of adults with anaplastic astrocytoma that has relapsed after treatment with a nitrosourea (lomustine or carmustine) and procarbazine.

The antibiotic aztreonam is now available as an inhalation solution (Cayston – Gilead) to improve respiratory symptoms in cystic fibrosis (CF) patients ≥7 years old colonized with Pseudomonas aeruginosa. It is the second inhaled antibiotic to be FDA-approved for this indication in CF patients; the aminoglycoside tobramycin (Tobi) was the first. Inhaled antibiotics offer the advantage of high airway concentrations while minimizing systemic side effects.