Pfizer's oral JAK inhibitor Litfulo (ritlecitinib) met co-primary endpoints in two Phase III trials (TRANQUILLO and TRANQUILLO 2) for active and stable nonsegmental vitiligo, with both 50 mg and 100 mg doses showing statistically significant, clinically meaningful benefit versus placebo at 52 weeks. Pfizer plans global regulatory submissions to seek approval of Litfulo as an oral systemic therapy for adults with NSV. Additional trial data will be presented at an upcoming medical meeting, while competitor JAK and topical therapies already hold recent approvals in NSV.
Pfizer Seeks Global Approval For Litfulo After Two Positive Phase III Trials In Nonsegmental Vitiligo
Pfizer will speak with regulatory authorities about the drug's approval in NSV. Credit: Poetra.RH / Shutterstock.com
Pfizer said its oral JAK inhibitor Litfulo (ritlecitinib) could be headed for a label expansion after meeting co-primary endpoints in two Phase III trials that evaluated treatment of both active and stable nonsegmental vitiligo (NSV).
Trial Program And Key Outcomes
The clinical program included TRANQUILLO (NCT05583526), which enrolled patients aged 12 years and older, and TRANQUILLO 2 (NCT06072183), which enrolled adults only. In both studies, Litfulo at 50 mg and 100 mg doses produced statistically significant and clinically meaningful improvements versus placebo on the co-primary measures: the facial Vitiligo Area Scoring Index (F-VASI) and the total-body Vitiligo Area Scoring Index (T-VASI).
Across the two trials, a significantly higher proportion of patients treated with Litfulo achieved F-VASI75 (>=75% improvement in facial VASI) and T-VASI50 (>=50% improvement in total VASI) at 52 weeks compared with placebo.
Endpoints And Regional Framework
The trials used slightly different endpoint frameworks by region: in the United States the co-primary endpoints were the proportions of patients reaching F-VASI75 and T-VASI50, while in other countries F-VASI75 served as the primary endpoint and T-VASI50 was a key secondary endpoint. Pfizer said it intends to submit global regulatory applications seeking approval of Litfulo as an oral systemic therapy for adults with NSV.
Context And Competitive Landscape
Litfulo was previously approved by the US Food and Drug Administration (FDA) in 2023 for severe alopecia areata in adults and adolescents aged 12 years and older. Pfizer also plans to initiate a pivotal study in 2026 to evaluate the drug for moderate alopecia areata.
These positive vitiligo results arrive amid recent competitor activity: AbbVie’s JAK inhibitor Rinvoq (upadacitinib) received European Commission approval for both NSV and alopecia areata, and Incyte’s topical ruxolitinib cream Opzelura holds approvals for NSV in the US and EU.
Disease Burden And Next Steps
Nonsegmental vitiligo is a chronic autoimmune condition in which immune-mediated destruction of melanin-producing cells leads to progressive loss of pigment, producing white macules and patches on skin and hair. NSV can begin at any age, affects all skin types, and often involves highly visible areas such as the face, neck and hands, which can have profound psychosocial impact.
Dr Iltefat Hamzavi, senior staff physician in the Department of Dermatology at Henry Ford Health and Hamzavi Dermatology Specialists, commented: "Living with NSV can have a profound impact on patients' daily lives. Many patients experience frustration due to the unpredictable nature of NSV as well as the limitations of currently available treatments and are seeking additional options that can address the underlying disease."
Additional detailed data from TRANQUILLO and TRANQUILLO 2 will be presented at an upcoming medical meeting. According to a GlobalData analysis, the prevalence of NSV across the 16 major pharmaceutical markets is projected to reach about 35 million cases by 2029.
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