Pfizer announced positive topline phase 3 results showing once-daily ritlecitinib (LITFULO) met co-primary endpoints for facial and total repigmentation versus placebo at week 52 in patients with nonsegmental vitiligo (NSV), across the TRANQUILLO and TRANQUILLO 2 trials.1
Ritlecitinib, an oral inhibitor of Janus kinase 3 (JAK3) and TEC family kinases, already carries FDA approval for severe alopecia areata in patients 12 years and older.2 No oral systemic therapy is currently approved specifically for nonsegmental vitiligo, leaving clinicians reliant on topical, phototherapy, and off-label systemic options.1 Based on the topline results, Pfizer intends to pursue global regulatory filings for ritlecitinib as a new oral systemic option for adults with NSV.1
"Living with nonsegmental vitiligo can have a profound impact on patients' daily lives," said Iltefat Hamzavi, MD, Senior Staff Physician, Department of Dermatology, Henry Ford Health, and Hamzavi Dermatology Specialists. "In these trials, ritlecitinib demonstrated significant improvements in both facial and total body repigmentation, reinforcing its potential to help support a new treatment paradigm with systemic therapies for patients whose disease burden warrants more than localized treatment."1
What Did TRANQUILLO Trials Show About Ritlecitinib Efficacy?
The phase 3 TRANQUILLO program included 2 pivotal trials plus a long-term extension study, TRANQUILLO LTE, for participants completing the parent TRANQUILLO trial.1 TRANQUILLO enrolled 607 adults and adolescents 12 years and older on 50 mg ritlecitinib once daily, while TRANQUILLO 2 enrolled 1,567 adults on 50 mg or 100 mg once daily, with 50 mg comparisons designated exploratory and descriptive.1 Combined, the trials evaluated 2,174 patients with active or stable NSV across 271 sites worldwide.1
In the United States, co-primary endpoints for both trials were the proportion of patients achieving F-VASI75 (≥75% improvement in Facial Vitiligo Area Scoring Index) and T-VASI50 (≥50% improvement in Total Vitiligo Area Scoring Index) at week 52.1 Outside the US, F-VASI75 at week 52 served as the primary endpoint, with T-VASI50 at week 52 as a key secondary endpoint.1 Pfizer reported significantly more patients receiving ritlecitinib achieved both F-VASI75 and T-VASI50 versus placebo at week 52 across both trials, though exact response rates and p-values were not disclosed in the topline release.1
Key Facts
What is ritlecitinib approved for?
Ritlecitinib (LITFULO) is FDA-approved for severe alopecia areata in adults and adolescents 12 years and older; it is not yet approved for nonsegmental vitiligo.
How does ritlecitinib work?
Ritlecitinib is an oral inhibitor of Janus kinase 3 (JAK3) and TEC family kinases, targeting immune signaling pathways implicated in immune cell attack on melanocytes and hair follicles.
What did the TRANQUILLO trials show?
TRANQUILLO and TRANQUILLO 2 met co-primary endpoints, showing significantly more patients receiving ritlecitinib achieved facial and total body repigmentation versus placebo at week 52; Pfizer has not yet disclosed exact response rates or p-values.
What is Ritlecitinib’s Safety Profile in Nonsegmental Vitiligo?
Pfizer characterized the safety profile of ritlecitinib in NSV as consistent with its established profile in severe alopecia areata, with no new safety signals identified.1 The proportion of patients experiencing treatment-emergent adverse events was reported as similar across all treatment groups, though the release did not provide specific incidence rates by arm.1 Ritlecitinib carries a boxed warning common to Janus kinase (JAK) inhibitors, including risks of serious infection, malignancy, major cardiovascular events, and thrombosis, particularly in patients 50 years and older with cardiovascular risk factors.2
Additional results from TRANQUILLO and TRANQUILLO 2, including detailed efficacy rates and statistical comparisons, are expected at a future medical meeting.1 The topline announcement did not include prespecified subgroup analyses by baseline disease severity or facial involvement, both cited by Pfizer as part of the trial populations.1
"LITFULO was internally discovered by Pfizer and has a unique mechanism of action targeting TEC family kinases and JAK3, giving rise to its distinct clinical profile," said Michael Vincent, MD, PhD, Chief Inflammation & Immunology Officer, Pfizer. "These findings give us confidence LITFULO could become a new oral systemic treatment option for adults living with NSV, significantly improving and potentially maintaining facial and total body repigmentation, if approved."1
The ritlecitinib development program spans alopecia areata, NSV, chronic spontaneous urticaria, and hidradenitis suppurativa.1 Pfizer also plans to initiate a pivotal study evaluating ritlecitinib for moderate alopecia areata in 2026.1
References
Pfizer Inc. Pfizer announces positive topline phase 3 results for LITFULO in patients with nonsegmental vitiligo. Published July 30, 2026. Accessed July 30, 2026. https://www.businesswire.com/news/home/20260730163060/en/Pfizer-Announces-Positive-Topline-Phase-3-Results-for-LITFULO-in-Patients-with-Nonsegmental-Vitiligo
LITFULO (ritlecitinib) capsules, for oral use. Prescribing information. New York, NY: Pfizer Inc; 2026. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/215830s001lbl.pdf