The European Commission (EC) has granted a marketing authorization for Incyte’s Opzelura (ruxolitinib) cream 15mg/g for the treatment of non-segmental vitiligo with facial involvement in adults and adolescents from 12 years of age.
Opzelura is the first and only approved treatment in the European Union (EU) to offer support for repigmentation in eligible patients with non-segmental vitiligo, a chronic autoimmune disease characterized by depigmentation of skin and reduced quality of life.
The EC decision follows the positive opinion received from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) earlier this year and is applicable to all 27 European Union Member States, Iceland, Norway and Liechtenstein.
“The approval of Opzelura by the European Commission represents a significant advancement for people living with non-segmental vitiligo with facial involvement who, until now, had no approved medical treatment to address repigmentation,” said Hervé Hoppenot, Chief Executive Officer, Incyte. “This approval wouldn’t have been possible without the support of the vitiligo patient and medical community, and the efforts of our research and development teams. We will now work across individual countries in Europe to bring this much awaited therapy to eligible patients seeking to treat their vitiligo.”
The EC decision is based on data from two pivotal Phase 3 clinical trials (TRuE-V1 and TRuE-V2), evaluating the efficacy and safety of Opzelura versus vehicle (non-medicated cream) in more than 600 people with non-segmental vitiligo, age 12 and older. Results from the TRuE-V program showed that treatment with Opzelura resulted in significant improvements in facial and total body repigmentation versus vehicle as shown by the number of patients reaching the facial and total body Vitiligo Area Scoring Index (F-VASI-T-VASI) endpoints at Week 24 compared to vehicle and in an open-label extension at Week 52.
Results at Week 24, which were consistent across both studies, showed that 29.8% and 30.9% of patients treated with Opzelura achieved ≥75% improvement from baseline in the facial Vitiligo Area Scoring Index (F-VASI75), the primary endpoint, compared to 7.4% and 11.4% of patients treated with vehicle in TRuE-V1 and TRuE-V2, respectively. At Week 52, approximately one in two of Opzelura-treated patients achieved F-VASI75.
Additionally, at Week 24, more than 15% of patients treated with Opzelura achieved ≥90% improvement from baseline in F-VASI (F-VASI90), compared to approximately 2% of patients treated with vehicle. At Week 52, approximately one in three of Opzelura-treated patients achieved F-VASI90.
There were no serious treatment-related adverse events related to ruxolitinib cream and the most common adverse reaction was application site acne.
Pivotal Phase 3 data supporting the European Commission decision were also published in The New England Journal of Medicine.
“The approval of Opzelura is welcome news for dermatologists and eligible patients in Europe who often face challenges managing vitiligo,” said Prof. Dr. Markus Böhm, Department of Dermatology, University of Münster Germany. “TRuE-V is the first large-scale clinical trials program in vitiligo and its results clearly demonstrate the clinically meaningful improvements in facial and total body repigmentation seen with Opzelura and its potential to further optimize patient care and outcomes.”
Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin that results in patchy loss of skin color from the progressive destruction of pigment-producing cells known as melanocytes. Over-activity of the JAK signaling pathway is believed to drive inflammation involved in the pathogenesis and progression of vitiligo. In the European Union, EEA countries and UK diagnosed vitiligo prevalence is estimated to be ~1.5 million patients, with approximately 8 in 10 patients suffering from non-segmental vitiligo, and a subset of whom has facial involvement and seeking treatment. Vitiligo can occur at any age, although many patients with vitiligo will experience initial onset before the age of 30.
“Vitiligo is a chronic autoimmune disease that impacts many aspects of a person’s life,” said Jean-Marie Meurant, Vice-President of the Vitiligo International Patient Organizations Committee (VIPOC). “The arrival of new treatment options are important for our community as it gives people with vitiligo something they have long been hoping for: the choice to treat their disease.”