Two new extended-release amphetamine products have been approved by the FDA for once-daily treatment of attention-deficit/hyperactivity disorder (ADHD) in patients ≥6 years old: Adzenys XR-ODT (Neos Therapeutics), an orally distintegrating tablet, and Dyanavel XR (Tris Pharma), an oral suspension.

Deflazacort (Emflaza – PTC Therapeutics), an oral corticosteroid, has been approved by the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients ≥5 years old. It has been available outside the US for many years. Deflazacort is the second drug to be approved for treatment of DMD; eteplirsen (Exondys 51), an antisense oligonucleotide approved for IV administration in patients with mutations of the dystrophin gene amenable to exon 51 skipping (about 13% of DMD cases), was the first.

The FDA has approved an oral powder formulation of the amino acid L-glutamine (Endari – Emmaus) to reduce the acute complications of sickle cell disease in patients ≥5 years old. Endari is only the second drug to be approved for use in sickle cell disease; the antimetabolite hydroxyurea (Droxia) was approved 20 years ago. L-glutamine oral powder is also available by prescription as NutreStore for treatment of short bowel syndrome and over the counter as a dietary supplement.

The FDA has approved hydrogen peroxide 40% topical solution (Eskata – Aclaris Therapeutics) for treatment of raised seborrheic keratoses (SKs) in adults. It is the first drug to be approved for this indication. (Hydrogen peroxide is available over the counter for topical use as a 3% solution.)

The oral kinase inhibitors dabrafenib (Tafinlar – GSK) and trametinib (Mekinist – Novartis) have received accelerated approval by the FDA for use together for a fifth indication: treatment of unresectable or metastatic solid tumors with a BRAF V600E mutation in patients ≥6 years old who have progressed following prior treatment and have no satisfactory alternative treatment options. The combination is not approved for treatment of colorectal cancer because of known intrinsic resistance to BRAF inhibition and dabrafenib is not approved for use in patients with wild-type BRAF...

Trofinetide (Daybue – Acadia), a synthetic analog of glycine-proline-glutamate, has been approved by the FDA for treatment of Rett syndrome in patients ≥2 years old. It is the first drug to be approved in the US for treatment of Rett syndrome.

A topical formulation of the antimetabolite mitomycin (Mitosol – Mobius Therapeutics) has been approved by the FDA for adjunctive use in glaucoma surgery to reduce scarring. Compounding pharmacies have supplied the drug off-label for this purpose for many years.

On May 10, 2021, the FDA expanded its Emergency Use Authorization (EUA) for the Pfizer/BioNTech mRNA-based COVID-19 vaccine to include adolescents 12-15 years old.1The vaccine has been authorized for use in persons ≥16 years old since December 2020.

Amlodipine (am loe' di peen) besylate (Norvasc - Pfizer), a dihydropyridine calcium-channel blocker, has been approved by the US Food and Drug Administration for once-daily oral treatment of hypertension, chronic stable angina, and vasospastic (Prinzmetal's) angina.

Echinacea, a plant also called purple coneflower, is widely used as a dietary supplement for prevention and treatment of colds.