Eli Lilly and Company wins FDA pediatric Olumiant approval for severe alopecia

A new U.S. treatment option for adolescents with severe alopecia areata opened Friday after the Food and Drug Administration expanded the Olumiant label for Eli Lilly and Company NYSELLY to patients aged 12 and older. In the pivotal trial, 42 per cent on the higher dose reached the scalp hair endpoint at 36 weeks, against 5 per cent on placebo.

The September 2026 prescribing label recommends 2 mg once daily, with an increase to 4 mg if response is inadequate. It says clinicians should consider starting at 4 mg when scalp hair loss is nearly complete or complete, then reduce the dose after an adequate response.

Lilly is a large-cap Indianapolis drugmaker that sells prescription medicines in the United States and overseas.

Lilly's NYSELLY quote showed shares at US$1,183.46 as of 4:03 p.m. EDT on Sept. 25. That quote preceded the after-hours announcement, so Friday's regular-session change does not measure investors' response to the approval.

A Pediatric Trial With a Scalp Hair Endpoint

BRAVE-AA-PEDS randomized 257 adolescents among placebo and two active-dose arms, Lilly said in its Sept. 25 release. Participants were aged 12 to under 18, and the active doses were 2 mg and 4 mg. The primary endpoint was a Severity of Alopecia Tool score of 20 or less at Week 36, meaning at least 80 per cent scalp hair coverage. The median score at entry was 100, indicating complete scalp hair loss.

The lower-dose group reached that endpoint in 27 per cent of patients, compared with 5 per cent on placebo. At the stricter threshold of 90 per cent scalp hair coverage, 37 per cent on 4 mg qualified. The 2 mg rate was 21 per cent, versus 2 per cent for placebo.

A second cohort of 166 adolescents received one of the active doses to build the safety database. Lilly described the pediatric safety profile as consistent with that in adults with severe alopecia areata, although the FDA label retains boxed warnings for serious infections, mortality, malignancy, major cardiovascular events and thrombosis. Lilly's release did not give a p value or confidence interval alongside the primary comparison.

"The evidence supporting Olumiant's approval is particularly meaningful because it comes from a Phase 3 study specifically designed for pediatric patients, helping clinicians and families make more informed decisions when considering advanced therapies," Yale School of Medicine adjunct associate professor of dermatology Brittany Craiglow said in Lilly's release.

The approval covers severe disease from age 12, not children younger than 12 or patients with milder alopecia areata. Lilly says the medicine is available through specialty pharmacies, but Lilly's Sept. 25 release mentions coverage at three large pharmacy benefit managers for adults with severe disease, without specifying pediatric coverage. It gives no pediatric reimbursement decision or sales forecast.

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Pfizer Entered the Adolescent Market Earlier

Pfizer Inc. NYSEPFE won U.S. approval for Litfulo, another oral treatment for severe alopecia areata from age 12, on June 23, 2023, according to its release. Pfizer reported that 23 per cent of Litfulo 50 mg recipients reached at least 80 per cent scalp hair coverage at Week 24. The placebo rate was 1.6 per cent. Different populations and follow-up periods prevent a direct comparison with Lilly's adolescent results at 36 weeks.

In its Sept. 25 release, Lilly estimates that as many as 2 per cent of people develop alopecia areata during their lives, with nearly 40 per cent of cases occurring before age 20. Those broad figures include milder cases and do not count U.S. adolescents eligible for the expanded Olumiant label.

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Lilly's third-quarter earnings call is scheduled for Oct. 29 at 10 a.m. EDT.