Afrezza Pediatric Approval Shows How FDA Requires and Rewards Children's Drug Studies

FDA has approved Afrezza, MannKind's inhaled mealtime insulin, for children and adolescents aged 6 and older with type 1 or type 2 diabetes, Managed Healthcare Executive reported on May 29, 2026. The product had been approved only for adults since June 2014. FDA's Drugs@FDA record lists the action as a supplement to the application, dated May 29, 2026.

The pediatric label did not arrive by chance. It is the end of a study FDA required when it first approved the drug, under a federal law that obliges many drug makers to test their products in children. A second law offers a reward for pediatric studies that companies agree to run voluntarily.

The study FDA required in 2014

The Pediatric Research Equity Act, or PREA, applies to applications for new active ingredients, new indications, new dosage forms, new dosing regimens or new routes of administration. FDA's 2014 approval letter for Afrezza says such applications must contain an assessment of safety and effectiveness in pediatric patients "unless this requirement is waived, deferred, or inapplicable."

For Afrezza, FDA did both. The letter waived studies for ages 0 to 3 because they were "impossible or highly impracticable," explaining that the inhaler was not appropriate for that age range. It deferred studies for ages 4 to 17 because the product was ready for approval in adults and the pediatric study had not been completed. The deferred study became a required postmarketing study, with its status to be reported to FDA every year. MannKind's 2014 timetable, quoted in the letter, set a final report date of January 2021. The letter also told the company to submit the results as a supplement with the labeling changes it believed the data supported.

That is the route the new approval took. According to Managed Healthcare Executive, the approval rests on INHALE-1, a 26-week, open-label Phase 3 trial in children and adolescents aged 4 to 17 that compared Afrezza plus basal insulin with multiple daily injections plus basal insulin. The primary endpoint was a noninferior change in HbA1c. The publication said the results were released in December 2024 and that Afrezza was noninferior in a modified analysis that excluded one participant who did not follow the protocol. The trial results are company-reported. The revised label, dated May 2026, says safety and effectiveness "have not been established in pediatric patients less than 6 years of age."

Waivers, deferrals and orphan drugs

FDA's 2023 draft guidance on pediatric drug development sets out how these decisions are made. A deferral can be granted when, among other grounds, a drug is ready for approval in adults before pediatric studies are complete. Sponsors must submit an initial pediatric study plan while the drug is still under investigation, so that children are considered early in development. PREA generally does not apply to a drug for an indication that has orphan designation, and for some new cancer drugs aimed at molecular targets relevant to childhood cancers, the law requires a separate pediatric investigation.

The voluntary route and its reward

The Best Pharmaceuticals for Children Act, or BPCA, works differently. FDA can issue a sponsor a Written Request describing pediatric studies it wants, if it determines that information about the drug's use in children may produce health benefits in that population, the guidance says. Sponsors may decline. Those that complete the requested studies may qualify for six months of additional marketing exclusivity, and the guidance says studies required under PREA can also earn that exclusivity if they are described in a Written Request issued before the sponsor submits its reports.

The guidance says the two laws are "designed to work together" to produce data on how drugs work in children. The FDA records and coverage reviewed for this article do not indicate whether Afrezza's pediatric studies were part of a Written Request.