Pfizer reports positive Phase 3 findings for LITFULO in patients with nonsegmental vitiligo

Pfizer has recently announced positive topline results from two Phase 3 trials assessing the efficacy and safety of once-daily LITFULO in patients with both active and stable nonsegmental vitiligo across a wide range of disease severity. The TRANQUILLO study recruited patients aged 12 and older, while TRANQUILLO 2 was limited to adults. Across both trials, LITFULO at both 50 mg and 100 mg doses delivered statistically significant and clinically meaningful gains over placebo on co-primary endpoints measuring facial and total body repigmentation using the Vitiligo Area Scoring Index. On the strength of these outcomes, Pfizer intends to submit global regulatory filings for LITFULO as a potential new oral systemic therapy for nonsegmental vitiligo in adults.

“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, M.D., Ph.D., Chief Inflammation and Immunology Officer, Pfizer. “The positive results from the TRANQUILLO pivotal studies in nonsegmental vitiligo build on our deep expertise in dermatology and on our experience with LITFULO in severe alopecia areata, including its established efficacy and well-characterized safety profile. These findings give us confidence that, if approved, LITFULO could become a new oral systemic treatment option for adults living with NSV, significantly improving and potentially maintaining facial and total body repigmentation.”

Nonsegmental vitiligo is a chronic autoimmune condition in which the immune system targets the cells responsible for producing melanin, stripping the skin and hair of pigment and giving rise to white patches and macules over time. It can develop at any age and across all skin types, with depigmentation capable of appearing anywhere on the body and frequently affecting highly visible areas including the face, neck, and hands. Although vitiligo is often dismissed as a cosmetic concern, it is a genuine autoimmune disease with a significant impact on patients’ lives that extends well beyond visible symptoms. Because the condition is chronic by nature, systemic treatment approaches may be necessary to help patients achieve and sustain repigmentation over the longer term.

Across both trials, a substantially greater proportion of patients receiving LITFULO achieved the threshold improvements in both facial and total body Vitiligo Area Scoring Index from baseline at week 52 compared with those on placebo. In the United States, the co-primary endpoints were the proportion of patients achieving at least 75% improvement in the facial score and at least 50% improvement in the total score at week 52. Outside the US, the facial improvement threshold served as the primary endpoint, with the total body improvement measure as a key secondary endpoint.

The safety profile of LITFULO in nonsegmental vitiligo remained consistent with what has previously been established in alopecia areata, with no new signals identified. The proportion of patients reporting treatment-emergent adverse events was broadly comparable across all groups.

“Living with nonsegmental vitiligo can have a profound impact on patients’ daily lives. Many patients experience frustration due to the unpredictable nature of nonsegmental vitiligo as well as the limitations of currently available treatments and are seeking additional options that can address the underlying disease,” said Iltefat Hamzavi, M.D., Senior Staff Physician, Department of Dermatology, Henry Ford Health and Hamzavi Dermatology Specialists. “In these trials, LITFULO demonstrated significant improvements in both facial and total body repigmentation, with the findings reinforcing the potential of LITFULO to help support a new treatment paradigm with systemic therapies for patients for whom disease burden warrants more than localized treatment alone.”

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Related Topics and Keywords

litfulo, nonsegmental vitiligo, Pfizer, Phase 3