Pfizer’s LITFULO (ritlecitinib) met Phase 3 endpoints in nonsegmental vitiligo, significantly improving facial and total body re-pigmentation versus placebo.
Written By: Shaik Yasmeen, PharmD
Reviewed By: Pharmacally Editorial Team
Pfizer has reported positive topline results from two pivotal Phase 3 clinical trials evaluating LITFULO (Ritlecitinib) for nonsegmental vitiligo (NSV), with both studies meeting their co-primary efficacy endpoints at Week 52. Once-daily LITFULO significantly improved both facial and total body re-pigmentation compared with placebo, supporting Pfizer’s plans to submit global regulatory applications seeking approval of the oral therapy for adults with NSV.
The results come from the TRANQUILLO and TRANQUILLO 2 studies, which together enrolled 2,174 patients across 271 clinical sites worldwide, making this the largest Phase 3 clinical program evaluating an oral systemic therapy for nonsegmental vitiligo to date.
LITFULO Targets Immune Pathways Driving Vitiligo
Nonsegmental vitiligo is a chronic autoimmune disease in which immune cells attack melanocytes, the pigment-producing cells responsible for skin color. The resulting loss of pigmentation often affects highly visible areas such as the face, hands, and neck and can have substantial psychological and social consequences. Although topical therapies and phototherapy remain standard treatment options, many patients with extensive disease may require systemic therapies to restore and maintain re-pigmentation.
LITFULO is an oral inhibitor of Janus kinase 3 (JAK3) and TEC family kinases. By inhibiting immune signaling pathways involved in melanocyte destruction, LITFULO targets the autoimmune processes underlying nonsegmental vitiligo. The drug is already approved in the United States, European Union, United Kingdom, Canada, Japan, and China for the treatment of severe alopecia areata in adults and adolescents aged 12 years and older.
Pivotal Studies Demonstrated Significant Re-pigmentation
The Phase 3 TRANQUILLO trial (NCT05583526) evaluated 50 mg once-daily LITFULO in 607 adults and adolescents aged 12 years and older, while TRANQUILLO 2 (NCT06072183) enrolled 1,567 adults and evaluated 50 mg and 100 mg once-daily regimens, with analyses involving the 50 mg arm considered exploratory and descriptive. Although adolescents participated in TRANQUILLO, Pfizer said its planned global regulatory submissions will initially seek approval for adults with nonsegmental vitiligo.
Across both pivotal studies, significantly more patients treated with LITFULO achieved the U.S. co-primary efficacy endpoints at Week 52 compared with placebo:
- F-VASI75: At least 75% improvement from baseline in the Facial Vitiligo Area Scoring Index.
- T-VASI50: At least 50% improvement from baseline in the Total Vitiligo Area Scoring Index.
Outside the United States, F-VASI75 served as the primary endpoint, while T-VASI50 was evaluated as a key secondary endpoint.
The safety profile was consistent with the established safety profile of LITFULO in severe alopecia areata. The proportion of patients experiencing treatment-emergent adverse events was similar across treatment groups, and no new safety signals were identified.
Positive Results Build on Established Dermatology Experience
Michael Vincent, M.D., Ph.D., Pfizer’s Chief Inflammation & Immunology Officer, said the findings build on the company’s dermatology expertise and the established clinical experience with LITFULO in severe alopecia areata. He said the drug’s dual inhibition of JAK3 and TEC family kinases provides a distinct mechanism that could offer adults with nonsegmental vitiligo a new oral systemic treatment option capable of improving and maintaining re-pigmentation.
Iltefat Hamzavi, M.D., Senior Staff Physician in the Department of Dermatology at Henry Ford Health and Hamzavi Dermatology Specialists, said patients with nonsegmental vitiligo continue to face unpredictable disease progression and limited treatment options. He added that the Phase 3 findings support the expanding role of systemic therapies for patients whose disease burden extends beyond localized involvement.
Regulatory Filings Planned as Development Expands
Pfizer plans to submit global regulatory applications for LITFULO as a treatment for adults with nonsegmental vitiligo, based on results from the TRANQUILLO clinical development program, which evaluated both adults and adolescents across its pivotal studies. Detailed efficacy and safety findings from the TRANQUILLO and TRANQUILLO 2 trials will be presented at a future medical meeting.
Beyond vitiligo, Pfizer continues to evaluate LITFULO across several immune-mediated dermatologic diseases, including chronic spontaneous urticaria and hidradenitis suppurativa. The company also plans to initiate a pivotal trial of LITFULO in moderate alopecia areata during 2026, further expanding its dermatology development program.
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About the Writer
Shaik Yasmeen (LinkedIn) is a Pharm.D graduate with interests in clinical pharmacy, pharmacovigilance, and medical writing. She has gained experience through hospital clinical postings, patient case reviews, case presentations, and literature evaluation. Passionate about evidence-based healthcare, she is committed to creating accurate and engaging medical content while continuously expanding her professional knowledge.