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.

Qualaquin, the only formulation of quinine sulfate available in the US, is approved only for treatment of uncomplicated malaria, but most prescriptions for its use are written for treatment or prevention of nocturnal leg cramps. The FDA recently issued a warning about its safety.Between April 2005 and October 2008, 38 cases of serious or life-threatening adverse effects of quinine were reported to the FDA. Twenty-one of these patients had thrombocytopenia and required hospitalization. Two deaths were reported: one from hemolysis and the other from thrombotic thrombocytopenic purpura (TTP)....

A recent editorial in The New England Journal of Medicine commented on the negative results of a clinical trial (published in the same issue) of nesiritide, a drug that had been approved by the FDA in 2001 (conditionally approved by Health Canada in 2007) for relief of dyspnea in patients with acutely decompensated heart failure. The authors of the recent clinical trial concluded: "On the basis of these results, nesiritide cannot be recommended for routine use in the broad population of patients with acute heart failure."1 The editorialist lamented the inadequacy of the data that had led to...

The FDA has warned that the IV emulsion formulation of the substance P/neurokinin 1 (NK1) receptor antagonist rolapitant (Varubi) has been associated in postmarketing reports with serious hypersensitivity reactions including anaphylaxis and anaphylactic shock.1 Rolapitant was approved by the FDA as an oral tablet in 2015 for adjunctive prevention of delayed nausea and vomiting associated with cancer chemotherapy in adults2; the IV emulsion formulation of the drug was approved for the same indication in 2017.The reported hypersensitivity reactions occurred during or shortly after infusion of...

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.