Pfizer's Phase 3 data lifts Litfulo as promising vitiligo therapy

PFE said Phase 3 data support Litfulo (ritlecitinib) as a treatment for nonsegmental vitiligo. Detailed results from the TRANQUILLO and TRANQUILLO 2 trials were presented on October 2 at the European Academy of Dermatology and Venereology Congress in Vienna, after Pfizer reported in July that both studies met their co-primary endpoints at Week 52.

Litfulo is not a new drug: the once-daily oral JAK3 and TEC kinase inhibitor has been FDA-approved since 2023 for severe alopecia areata in patients 12 and older. The vitiligo program is a bid for a second indication. Across the two trials, 2,174 patients with active or stable disease were enrolled. TRANQUILLO tested 50 mg in 607 adults and adolescents, while TRANQUILLO 2 tested 50 mg or 100 mg in 1,567 adults.

The U.S. co-primary endpoints were the share of patients with at least 75% improvement in the facial Vitiligo Area Scoring Index (F-VASI75) and at least 50% improvement in the total-body index (T-VASI50) at Week 52. At 100 mg, 21.8% of patients reached F-VASI75 and 13% reached T-VASI50, versus about 2.4% on placebo for each. At 50 mg in TRANQUILLO, 12.4% reached F-VASI75 versus about 2.5% on placebo, and 8.9% reached T-VASI50 versus 1.9%. Pfizer said improvement appeared by Week 24 and continued through Week 52, and that safety was consistent with the alopecia areata profile with no new signals.

The absolute response rates are modest even where the gap to placebo is wide, which frames how the drug may be positioned. Pfizer intends to seek approval for adults with the FDA and the European Medicines Agency but has not given a filing date. The competitive field includes Incyte's topical Opzelura, which MedCity News describes as the only FDA-approved vitiligo drug, and AbbVie's oral Rinvoq, which added vitiligo to its European label in July and is under FDA review for the indication. Filing timing and the Rinvoq U.S. decision are the next markers to watch.