The tissue plasminogen activator (tPA) tenecteplase (TNKase – Genentech) has been approved by the FDA for treatment of acute ischemic stroke in adults. It is the second tPA to be approved in the US for this indication; alteplase (Activase) was approved in 1996. Tenecteplase was approved in 2000 to reduce the risk of death associated with acute ST-elevation myocardial infarction (STEMI).

Prademagene zamikeracel (Zevaskyn – Abeona Therapeutics), an autologous cell sheet-based gene therapy, has been approved by the FDA for treatment of wounds in patients with recessive dystrophic epidermolysis bullosa. It is the first autologous cell sheet-based gene therapy to be approved in the US for this indication. Beremagene geperpavek gel (Vyjuvek), a herpes simplex virus type 1 vector-based gene therapy, is approved for once-weekly use in patients ≥6 months old with recessive dystrophic epidermolysis bullosa.

Permethrin 5% (Elimite Cream - Herbert), a synthetic pyrethroid previously available in a 1% concentration (Nix) for treatment of head lice (Medical Letter, 28:89, 1986) and as an aerosol clothing spray (Permanone) for protection against mosquitoes and ticks (Medical Letter, 31:45, 1989), was recently approved by the US Food and Drug Administration for single-application treatment of infestation with Sarcoptes scabiei (scabies).

Patients may be asking about reports in the lay press that exenatide (Byetta - Med Lett Drugs Ther 2005; 47:45), a synthetic peptide that stimulates release of insulin from pancreatic beta cells, has been used by diabetics and non-diabetics to lose weight. Approved by the FDA to improve glycemic control in patients with type 2 diabetes not controlled by metformin, a sulfonylurea or both, it is given by subcutaneous injection before the morning and evening meals. In clinical trials, some diabetic patients treated with the drug lost weight. No data are available on use in non-diabetics....

Chelation therapy involves oral administration, intravenous infusion or intramuscular injection of drugs that increase excretion of heavy metals. The Medical Letter’s last article on this subject found no evidence that it was effective for treatment of cardiovascular disease. Since then, off-label use of chelation therapy has expanded to include treating children with autism and adults with Alzheimer’s disease, cancer and other chronic diseases.

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Dichlorphenamide (Keveyis – Taro), an oral carbonic anhydrase inhibitor, has been approved by the FDA for treatment of primary hypokalemic and hyperkalemic periodic paralysis and related variants. Dichlorphenamide is the first drug to be approved in the US for this indication. It was approved as Daranide in 1958 for treatment of glaucoma, but had not been marketed since 2002.

The FDA has approved the atypical antipsychotic pimavanserin (Nuplazid – Acadia) for treatment of hallucinations and delusions associated with Parkinson's disease. It is the first drug to be approved in the US for this indication.

A fixed-dose combination of orphenadrine citrate, aspirin, and caffeine (Orphengesic Forte – Galt; previously available as Norgesic Forte) has been approved as a prescription drug by the FDA for treatment of mild to moderate pain caused by acute musculoskeletal disorders. Single-ingredient generic orphenadrine citrate is available by prescription in oral and injectable formulations and has been used for years as an adjunct for treatment of acute musculoskeletal pain. Orphengesic Forte is being marketed as a non-opioid alternative for pain relief.

A recent article in JAMA and an interview of its senior author on 60 Minutes have heightened interest in off-label use of the oral selective serotonin reuptake inhibitor (SSRI) fluvoxamine (Luvox, and generics) to treat COVID-19.

A reader asked us to review use of the phosphodiesterase-5 (PDE5) inhibitor sildenafil for treatment of Alzheimer's disease (AD).