FDA批准巴瑞替尼用于12岁及以上青少年重度斑秃
Baricitinib Approved for Severe Alopecia Areata in Adolescents
FDA扩大礼来巴瑞替尼（Olumiant）标签，批准其用于12岁及以上重度斑秃青少年患者，此前该药仅获批用于成人。获批基于BRAVE-AA-PEDS 3期研究，36周时4mg剂量组42%患者达到SALT评分≤20（头皮覆盖率≥80%），而安慰剂组仅5%。该药是继利特昔替尼之后第二个获批用于该人群的口服系统疗法。
原文给出了巴瑞替尼获批用于青少年重度斑秃的适应症扩展和关键临床数据，有助于从业者对比已有疗法做出用药决策。
Baricitinib is now FDA approved for adolescents 12 and older with severe alopecia areata, extending the once-daily pill beyond adults.
Patients as young as 12 years old with severe alopecia areata (AA) can now be prescribed baricitinib (Olumiant; Eli Lilly), a once-daily oral systemic therapy previously approved only for adults with the condition.1 The FDA expanded the drug’s label to patients 12 years and older based on the results of the BRAVE-AA-PEDS study.
As many as 2% of people develop AA in their lifetimes, and nearly 40% of cases begin before age 20, with earlier onset often linked to more extensive hair loss.
“Today's approval marks an important milestone for young people living with severe alopecia areata and their families, who have had limited treatment options to date,” said Adrienne Brown, executive vice president and president, Lilly Immunology, in a statement. “The visible and often extensive hair loss that comes with severe alopecia areata can have a profound impact on anyone, especially adolescents who are at a pivotal stage of life.”
Baricitinib is the second systemic oral therapy approved for patients 12 years and older with severe AA. Ritlecitinib (Litfulo; Pfizer) received approval for the age group in June 2023.2
Why Alopecia Areata Weighs Heavily on Adolescents
Brittany Craiglow, MD, adjunct associate professor of dermatology, Yale School of Medicine, described AA in an interview with The American Journal of Managed Care® as more than just the loss of hair, calling it a “loss of identity” for people who look at themselves in the mirror and don’t see themselves as they expect to look.3
“AA is often considered to be just hair, but we know from the patient experience that it's really so much more than that,” Craiglow said. “We see increased rates of anxiety and depression.”
The Phase 3 Data Supporting Baricitinib’s Adolescent Approval
The 36-week results from the BRAVE-AA-PEDS trial, first presented at the 2025 American Academy of Dermatology (AAD) annual meeting,4 were the basis of the FDA approval. The adolescent study of patients aged 12 to under 18 years was described by Lilly as the first and largest phase 3 study designed specifically for pediatric patients with severe AA.
The study assigned 257 adolescents in randomized fashion 1:1:1 to receive placebo, baricitinib 4 mg, or baricitinib 2 mg.1 The median Severity of Alopecia Tool (SALT) score was 100, or complete scalp hair loss. The patients had lived with AA for a mean of 6.4 years.
At week 36, 42% of patients on 4 mg baricitinib and 27% on the 2 mg dose had achieved a SALT score of 20 or lower, which is equivalent to 80% or more scalp coverage, compared with just 5% on placebo. In addition, 37% on the 4 mg dose, 21% on the 2 mg dose, and 2% on placebo achieved a SALT score of 10 or lower, which is 90% or more scalp coverage. The patients on the 4 mg dose who had substantial eyebrow and eyelash loss at baseline also experienced improvements in that coverage.
“Severe alopecia areata that starts early in life can be more extensive and harder to treat over time, making it important to consider all available treatment options early in the course of disease,” Craiglow said in a statement. “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.”
The overall safety profile for the pediatric cohort was consistent with the adult cohort in earlier studies. Baricitinib carries a boxed warning for serious infections, mortality, malignancy, major adverse cardiovascular events, and thrombosis.
How Adult and Longer-Term Adolescent Data Compare
The 52-week data presented at the 2025 Fall Clinical Dermatology conference built on the 36-week data. More than half (54.1%) of adolescents on 4 mg achieved a SALT score of 20 or lower, and 41.2% achieved a score of 10 or lower.5 For the patients on the 2 mg dose, 31% achieved a score of 20 or lower and 26.2% a score of 10 or lower. Of the patients who had severe, but not total, hair loss (scores of 50 to 94), 71% on 4 mg achieved successful hair regrowth, which underscores the value of early intervention.
A 3-year analysis of the BRAVE-AA1 and BRAVE-AA2 trials in adults found that, of those who responded and stayed on the same dose, 89.1% of patients on 4 mg and 83.6% on 2 mg maintained a SALT score of 20 or lower at week 152.6 In a real-world study of 48 adults, 58.3% had reached a SALT score below 20, and 36.4% were classified as nonresponders.7
Approval of ritlecitinib for adolescents was based on results of the phase 2b/3 ALLEGRO trial of 718 adolescents and adults.2 Nearly a quarter (23%) of patients on the 50 mg dose reached a SALT score of 20 or lower after 6 months compared with 1.6% on placebo.
Ongoing Safety Cohort Study
BRAVE-AA-PEDS is ongoing with a second cohort of 166 adolescents, who were assigned in a randomized 1:1 fashion to receive either 4 mg baricitinib or 2 mg to accumulate more safety data.1
For adults, the 2 mg dose is recommended for baricitinib with an increase to 4 mg if treatment response is inadequate. Patients with complete or nearly complete scalp hair loss may consider the 4 mg dose, which is to be reduced to 2 mg once an adequate response has been reached.
“More treatment choices mean more families have the opportunity to find the option that's right for their child, together with their doctor,” George Gondo, chief mission officer at National Alopecia Areata Foundation, said in a statement.1
References
1. FDA approves Lilly's Olumiant (baricitinib) for pediatric patients 12 years of age and older with severe alopecia areata. News release. Eli Lilly. September 25, 2026. Accessed September 28, 2026. https://investor.lilly.com/news-releases/news-release-details/fda-approves-lillys-olumiant-baricitinib-pediatric-patients-12
2. FDA approves Pfizer’s Litfulo (ritlecitinib) for adults and adolescents with severe alopecia areata. News release. Pfizer. June 23, 2023. Accessed September 28, 2026. https://www.pfizer.com/news/press-release/press-release-detail/fda-approves-pfizers-litfulotm-ritlecitinib-adults-and
3. Jeremias S, Craiglow B. The psychiatric burden of pediatric alopecia areata: a Q&A with Brittany Craiglow, MD. AJMC®. August 3, 2026. Accessed September 28, 2026. https://www.ajmc.com/view/psychiatric-burden-of-pediatric-alopecia-areata-brittany-craiglow-md
4. Santoro C, Craiglow B. Baricitinib shows promise for pediatric alopecia in BRAVE-AA-PEDS study. AJMC. March 9, 2025. Accessed September 28, 2026. https://www.ajmc.com/view/baricitinib-shows-promise-for-pediatric-alopecia-in-brave-aa-peds-study
5. McNulty R. Baricitinib leads to significant hair regrowth in adolescents with severe alopecia areata. AJMC. November 5, 2025. Accessed September 28, 2026. https://www.ajmc.com/view/baricitinib-leads-to-significant-hair-regrowth-in-adolescents-with-severe-alopecia-areata
6. Jeremias S. Three years on baricitinib preserved hair regrowth in severe alopecia areata. AJMC. June 4, 2026. Accessed September 28, 2026. https://www.ajmc.com/view/three-years-on-baricitinib-preserved-hair-regrowth-in-severe-alopecia-areat
7. Grossi G. Baricitinib shows real-world effectiveness in severe alopecia areata hair loss. AJMC. December 24, 2025. Accessed September 28, 2026. https://www.ajmc.com/view/baricitinib-shows-real-world-effectiveness-in-severe-alopecia-areata-hair-loss
来源：AJMC 管理式医疗 · ajmc.com