Dordaviprone (Modeyso – Jazz), an oral protease activator, has received accelerated approval from the FDA for treatment of diffuse midline gliomas harboring a H3 K27M mutation in patients ≥ year old who had disease progression following prior treatment. It is the first drug to be approved in the US for this indication.

Saxagliptin (Onglyza - Bristol-Myers Squibb), the second oral dipeptidyl peptidase-4 (DPP-4) inhibitor to be marketed in the US, has been approved by the FDA for treatment of adults with type 2 diabetes.

The FDA has approved Akynzeo (Helsinn/Eisai), an oral fixed-dose combination of the substance P/neurokinin 1 (NK1) receptor antagonist netupitant and the serotonin-3 (5-HT3) receptor antagonist palonosetron, for prevention of acute and delayed nausea and vomiting associated with cancer chemotherapy in adults. Akynzeo is the first product to combine drugs from these two classes. Palonosetron (Aloxi) is also available as a single agent for prevention of chemotherapy-induced and postoperative nausea and vomiting. Netupitant is the second substance P/NK1 receptor antagonist to be...

The FDA has approved lemborexant (Dayvigo – Eisai), an orexin receptor antagonist, for treatment of sleep-onset and/or sleep-maintenance insomnia in adults. It is the second orexin receptor antagonist to be approved for this indication; suvorexant (Belsomra) was the first.

A Medical Letter reader asked if human chorionic gonadotropin (hCG; Novarel, Pregnyl and others) has any value as an adjunct to diet, adding that it is widely used for this indication in his area. Normally secreted by the placenta during pregnancy, the main therapeutic use of this gonad-stimulating polypeptide, which is not absorbed from the GI tract, has been in the parenteral treatment of infertility.1 It has also been injected by male athletes as an undetectable stimulus to production of testosterone.2Use of hCG as an adjunct to diet goes back to the 1950’s, when a British physician named...

The FDA has removed the Risk Evaluation and Mitigation Strategy (REMS) requirement for the endothelin receptor antagonists ambrisentan (Letairis, and generics), bosentan (Tracleer, and generics), macitentan (Opsumit, and generics; Opsynvi), and aprocitentan (Tryvio), and the endothelin receptor antagonist and angiotensin II receptor antagonist sparsentan (Filspari) that was initially implemented because of a possible risk of embryofetal toxicity.

Gonadorelin (goe nad oh rell'; in) acetate (Lutrepulse - Ortho), a synthetic decapeptide identical in amino acid sequence to human gonadotropin-releasing hormone (GnRH), was recently approved by the US Food and Drug Administration for treatment of primary hypothalamic amenorrhea. Gonadorelin hydrochloride (Factrel) was previously available for diagnostic use (Medical Letter, 25:106, 1983). Analogs of GnRH have been used to treat endometriosis and advanced cancer of the prostate (Medical Letter, 27:71, 1985).

The February 14th issue of The Medical Letter reviewed natalizumab (Tysabri - Biogen Idec), a new monoclonal antibody that was granted accelerated approval because it decreased the number of relapses in patients with multiple sclerosis. On February 28th, the FDA issued a Public Health Advisory announcing that marketing of the drug had been suspended because 2 patients who had been treated with natalizumab for more than 2 years had developed progressive multifocal leukoencephalopathy, a rare, often fatal disease...

The FDA issued a public health advisory on March 10, 2005 warning about a risk of cancer with topical formulations of tacrolimus (Protopic) and pimecrolimus (Elidel) used to treat eczema in adults and children more than 2 years old. Elidel has been heavily promoted to the general public on television. The warning was based on reports of dose-related cancer in animals and 29 reports of cancer (including 8 skin malignancies and 12 lymphomas) in adults and children treated with these immunosuppressive drugs. Cause and effect have not been established. When Protopic was first marketed, The Medical...

The standard antithrombotic therapy for treatment of patients with acute coronary syndrome (ACS) is dual antiplatelet therapy with aspirin and clopidogrel (Plavix) or another thienopyridine, plus a parenteral anticoagulant while the patient is hospitalized, followed by antiplatelet therapy alone after discharge. The addition of the oral anticoagulant warfarin (Coumadin, and others) to dual antiplatelet therapy is generally not recommended for this indication because of fluctuations in its anticoagulant effect and the risk of bleeding. A recently published trial found that addition of...