Pfizer is moving closer to expanding the use of its oral drug LITFULO (ritlecitinib) after two pivotal Phase 3 clinical trials met their primary goals in adults with nonsegmental vitiligo, a chronic autoimmune skin disorder with limited systemic treatment options.

 

The company said both the TRANQUILLO and TRANQUILLO 2 studies demonstrated statistically significant and clinically meaningful improvements in facial and total body repigmentation compared with placebo after 52 weeks of treatment. 

 

Based on the positive topline results, Pfizer plans to submit global regulatory applications seeking approval of LITFULO as an oral systemic treatment for adults with nonsegmental vitiligo.

 

The trials represent the largest Phase 3 clinical program to date evaluating an oral systemic therapy for the disease, enrolling a combined 2,174 patients across 271 sites worldwide. TRANQUILLO included patients aged 12 years and older, while TRANQUILLO 2 enrolled adults only.

 

"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, Chief Inflammation & 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."

 

Across both studies, significantly more patients receiving either the 50 mg or 100 mg dose of LITFULO achieved at least a 75% improvement in facial Vitiligo Area Scoring Index (F-VASI75) and a 50% improvement in total body Vitiligo Area Scoring Index (T-VASI50) compared with those receiving placebo at Week 52.

 

The drug's safety profile remained consistent with previous studies in alopecia areata, with no new safety signals identified. Overall rates of treatment-emergent adverse events were similar across treatment groups, Pfizer said.

 

Nonsegmental vitiligo is an autoimmune disease in which the immune system attacks pigment-producing melanocytes, leading to white patches on the skin and hair. Although often perceived as a cosmetic condition, the disease can have a significant psychological and social impact on patients and frequently requires long-term management.

 

"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. 

 

"In these trials, LITFULO (ritlecitinib) 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."

 

LITFULO is already approved in the United States and several other markets for the treatment of severe alopecia areata in adults and adolescents aged 12 years and older. The medicine works by inhibiting Janus kinase 3 (JAK3) and TEC family kinases, key immune signaling pathways involved in autoimmune diseases.