Pfizer has reported positive topline results from two Phase 3 trials evaluating LITFULO (ritlecitinib) in patients with nonsegmental vitiligo (NSV), supporting the company’s plans to pursue regulatory filings for the oral therapy as a potential systemic treatment for adults with the chronic autoimmune skin disorder.
The TRANQUILLO study enrolled patients aged 12 years and older, while TRANQUILLO 2 included adults. Both trials evaluated once-daily doses of LITFULO at 50 mg and 100 mg in patients with active and stable NSV covering a broad range of disease severity. According to Pfizer, both doses produced significant and clinically meaningful improvements compared with placebo on the studies’ key measures of facial and total-body repigmentation.
Based on the findings, Pfizer plans to submit regulatory applications in markets around the world seeking approval for LITFULO as a potential oral systemic therapy for adults with nonsegmental vitiligo.
Vitiligo is a chronic autoimmune disease in which the immune system attacks melanocytes, the cells responsible for producing the pigment melanin. This can result in white patches and macules appearing on the skin and hair. Nonsegmental vitiligo is the most common form of the disease and can affect people of any age and skin type. Depigmentation can occur across different parts of the body, with the face, neck and hands among the areas frequently affected.
Although vitiligo is sometimes viewed primarily as a cosmetic condition, the disease can have a substantial impact on patients’ emotional and social well-being. Because the condition is chronic and can progress unpredictably, patients may require treatments capable of restoring pigmentation and helping maintain those improvements over time.
In the two Pfizer trials, significantly more patients receiving LITFULO achieved predefined improvements in both facial and total-body repigmentation by Week 52 compared with those receiving placebo. The studies assessed the proportion of patients achieving at least a 75% improvement from baseline in the Facial Vitiligo Area Scoring Index, known as F-VASI75, as well as at least a 50% improvement in the Total Vitiligo Area Scoring Index, or T-VASI50.
For the US studies, F-VASI75 and T-VASI50 at Week 52 were the co-primary endpoints. Outside the US, F-VASI75 at Week 52 served as the primary endpoint, while T-VASI50 at the same time point was considered a key secondary endpoint.
Pfizer said the safety profile of LITFULO in the vitiligo studies was consistent with the treatment’s established safety profile in alopecia areata. No new safety signals were identified, and the overall proportion of patients experiencing treatment-emergent adverse events was similar among the treatment groups.
The results build on Pfizer’s existing experience with LITFULO, which was internally discovered by the company and has a mechanism targeting TEC family kinases and JAK3. The drug is already approved in the US for the treatment of severe alopecia areata in adults and adolescents aged 12 years and older.
Michael Vincent, Pfizer’s Chief Inflammation and Immunology Officer, said the positive TRANQUILLO results add to the company’s experience with LITFULO in dermatology and its established efficacy and safety profile in severe alopecia areata. He said the findings provide confidence in the potential for LITFULO to become an oral systemic treatment option for adults with NSV if approved.
The company’s decision to pursue regulatory submissions reflects the potential need for additional systemic therapies in nonsegmental vitiligo, particularly for patients whose disease affects extensive or highly visible areas of the body. Current approaches can include localized therapies, but patients with more widespread disease may require treatment capable of addressing pigmentation loss across larger areas.
Dermatologist Iltefat Hamzavi, who participated in the clinical development program, said the disease can have a significant effect on daily life and that many patients continue to seek additional treatment options. He said the Phase 3 findings demonstrated improvements in both facial and total-body repigmentation and could support a greater role for systemic therapies in appropriate patients.
Pfizer plans to present additional results from the TRANQUILLO and TRANQUILLO 2 studies at a future medical meeting. Further data from the trials will provide additional information about the efficacy and safety of LITFULO in different patient groups and disease profiles.
LITFULO’s potential expansion into vitiligo would broaden the commercial and therapeutic role of the medicine beyond alopecia areata. Pfizer also plans to begin a pivotal study in 2026 evaluating LITFULO in moderate alopecia areata, further expanding development of the drug across dermatological conditions.
If approved for nonsegmental vitiligo, LITFULO could provide patients with an oral systemic treatment option designed to promote repigmentation across both the face and broader areas of the body. The next stages of development will focus on regulatory review and the submission of additional clinical data as Pfizer works toward potential approvals in the US and other global markets.