Pfizer has announced positive topline results from two phase 3 trials evaluating the efficacy and safety of Litfulo once daily in patients with both active and stable nonsegmental vitiligo (NSV) and who had a broad range of disease severity.
The TRANQUILLO study included patients aged 12 years and older, while TRANQUILLO 2 enrolled adults only. Across the studies, both the 50 and 100 milligram doses of Litfulo delivered significant, clinically meaningful improvements over placebo on co-primary endpoints for the facial and total body Vitiligo Area Scoring Index, or VASI.
Based on these results, Pfizer intends to submit global regulatory filings for Litfulo as a potential new oral systemic therapy for NSV for adults.
“Litfulo has a unique mechanism of action targeting TEC family kinases and JAK3, giving rise to its distinct clinical profile,” said Michael Vincent, Chief Inflammation & Immunology Officer at Pfizer. “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.”
NSV is a chronic autoimmune disease in which the immune system attacks melanin-producing cells, leading to a loss of pigment and resulting in white macules and patches on the skin/hair over time. NSV can appear at any age and in all skin types, causing depigmentation anywhere on the skin, often affecting highly visible areas such as the face, neck, and hands. While vitiligo is frequently treated as a cosmetic condition, it is a serious autoimmune disease that can have a considerable impact on patients’ lives well beyond the physical symptoms. Because NSV is a chronic disease, systemic treatment options may be needed to help patients restore skin pigmentation and maintain repigmentation over time.
“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, Senior Staff Physician, Department of Dermatology, Henry Ford Health and Hamzavi Dermatology Specialists.