FDA Actions
The FDA has approved edaravone for the treatment of amyotrophic lateral sclerosis (ALS), the first new treatment for ALS in more than 20 years. The drug is a potent radical scavenger, which was developed in Japan to treat acute cerebral embolism. Efficacy for ALS was shown in 137 patients given edaravone or placebo; edaravone-treated patients showed modest improvement in the ALS functional rating scale up to week 24. The drug is given as an IV infusion daily for 14 days, followed by a 14-day drug-free period, then for 10 days every 28 days. The cost is $1,000 per daily dose. Edaravone was approved with orphan designation and will be marketed starting in August as Radicava.
The FDA has expanded the indication for the checkpoint inhibitor pembrolizumab (Keytruda) to include all solid tumors with a specific biomarker, the first time the agency has approved a cancer treatment based on a biomarker rather than the location in the body where the tumor originated. Checkpoint inhibitors, including pembrolizumab, target the programmed cell death 1 (PD-1/PD-L1) receptor on cancer cells. The specific biomarker covered under this new approval is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). With the new approval, pembrolizumab is approved for adult and pediatric patients with unresectable or metastatic solid tumors, most commonly colorectal, endometrial, and gastrointestinal cancers, but also less frequently in breast, prostate, bladder, thyroid gland, and others. The accelerated approval was based on data from 149 patients with MSI-H or dMMR cancers enrolled across five single-arm clinical trials. Pembrolizumab previously was approved for certain patients with metastatic melanoma, metastatic non-small cell lung cancer, recurrent or metastatic head and neck cancer, refractory classical Hodgkin lymphoma, and urothelial carcinoma.
The FDA has expanded the indication for tocilizumab to include giant cell arteritis (GCA). The drug is an interleukin-6 receptor antagonist that previously was approved for rheumatoid arthritis. It is given as a weekly subcutaneous injection. Efficacy and safety were shown in a study of 251 patients with GCA treated with tocilizumab plus prednisone vs. prednisone plus placebo, in which combination therapy resulted in a higher number of patients with sustained remission. The drug carries a boxed warning regarding severe infections. Tocilizumab was granted breakthrough therapy designation and given a priority review. It is marketed as Actemra.
The FDA has approved the first generic version of the attention-deficit/hyperactivity disorder (ADHD) drug atomoxetine (Strattera). Four manufacturers are ready to start production immediately. Atomoxetine is approved for the treatment of ADHD in pediatric and adult patients. Like other ADHD medications, atomoxetine carries a boxed warning for the increased risk of suicidal ideation in children and adolescents.
In this section: FDA approves new amyotrophic lateral sclerosis, expands the indication for pembrolizumab, adds giant cell arteritis to the indication for tocilizumab, and green lights generic ADHD drug.
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