The US Food and Drug Administration (FDA) has approved 'early access' use of riluzole (Rilutek - Rh ne-Poulenc Rorer) for treatment of amyotrophic lateral sclerosis (ALS). is the first drug to become available for treatment of this condition. The premarketing supply is limited; the manufacturer (1-800-798-7425) has enough on hand to treat 3,000 of the 25,000 patients with the disease in the USA, and those 3,000 have already been selected through a lottery. Similar arrangements have been made in several European countries. Riluzole is not available commercially in any country.

The FDA has approved edaravone (Radicava – Mitsubishi Tanabe Pharma) for treatment of amyotrophic lateral sclerosis (ALS). It is the second drug to be approved in the US for this indication; riluzole (Rilutek, and generics) was approved in 1995. Edaravone was approved for treatment of ALS as Radicut in Japan and South Korea in 2015. It has been used in Japan and other Asian countries since 2001 for treatment of acute ischemic stroke.

Relyvrio, the fixed-dose combination of sodium phenyl-butyrate and taurursodiol that received accelerated approval for treatment of amyotrophic lateral sclerosis (ALS) in 2022 based on a phase 2 trial showing that it slowed functional decline, has voluntarily been withdrawn from the market. According to the manufacturer (Amylyx), the decision to withdraw Relyvrio was based on the results of a phase 3, 48-week trial (PHOENIX). The change from baseline on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale, which measures fine motor, gross motor, bulbar, and respiratory...

The FDA has approved several significant changes in the labeling of mifepristone (Mifeprex – Danco), an oral antiprogestin that has been used in the US for more than 15 years for termination of intrauterine pregnancy. It has generally been used with the prostaglandin analog misoprostol (Cytotec, and generics).

The FDA has approved spinosad 0.9% suspension (Natroba – ParaPro) for topical treatment of head lice infestation in patients ≥4 years old. It is available only by prescription.

The FDA has approved the antiparasitic drug ivermectin in a 0.5% lotion (Sklice – Sanofi Pasteur) as a single-use topical treatment for head lice in patients ≥6 months old. Oral ivermectin (Stromectol – Merck) is effective for treatment of head lice resistant to other therapies, but it has not been approved by the FDA for this indication.

The FDA has approved Relyvrio (Amylyx), a fixed-dose combination of sodium phenylbutyrate and taurursodiol, for treatment of amyotrophic lateral sclerosis (ALS). Sodium phenylbutyrate (Buphenyl, and others) has been available by prescription for years for treatment of urea cycle disorders. Taurursodiol (tauroursodeoxycholic acid), a derivative of ursodiol, is an over-the-counter bile acid supplement claimed to have neuroprotective benefits. Relyvrio is the third drug to be approved in the US for treatment of ALS; riluzole (Rilutek, and others) and edaravone (Radicava, Radicava...

Tofersen (Qalsody – Biogen), an intrathecally administered antisense oligonucleotide, has received accelerated approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. About 2% of patients with ALS have mutations in the SOD1 gene. Tofersen is the first drug to be approved in the US that targets a genetic cause of ALS. Accelerated approval of the drug was based on the surrogate endpoint of a reduction in plasma neurofilament light chain, which is considered likely to predict clinical...

View Figure 1: Pneumococcal Vaccine Recommendations for Adults 19-64 Years Old

0.5% malathion in 78% isopropanol was recently approved by the FDA for treatment of head lice.