Appeal type: Formulary Exception
Decision: Overturned

Diagnosis: Vitiligo
Treatment: Formulary exception for Opzelura 1.5% Cream
The insurer denied: Formulary exception for Opzelura 1.5% Cream
The denial is overturned

The patient is an adult male with vitiligo affecting 15% body surface area for whom Opzelura 1.5% cream is requested. He has been using Opzelura 1.5% topical cream twice daily for 2 months. He was also prescribed phototherapy at the time but has not initiated it yet. Medical records show that he has used mometasone cream, tacrolimus ointment, fluocinonide ointment, Elidel cream, hydrocortisone cream, triamcinolone cream, halobetasol propionate ointment and ketoconazole cream, with no relief.

The health plan should cover the Formulary exception for Opzelura.

The formulary drugs are all topical corticosteroids of varying potency (alclometasone dipropionate, betamethasone dipropionate, betamethasone valerate, clobetasol propionate, desonide, desoximetasone,diflorasone, fluocinolone acetonide, fluocinonide, fluticasone, halobetasol propionate, hydrocortisone, mometasone, triamcinolone acetonide), many of which the patient has already tried and failed. Trying other topical steroids will make no difference and can result in long-term adverse effects due to thinning of the skin or suppression of the HPA (hypothalamic-pituitary-adrenal) axis, since the patient has a large body surface area involved. Currently, there is no treatment that is FDA (United States Food and Drug Administration) approved for use in vitiligo.

OPZELURA (ruxolitinib) is used on the skin (topical) for short-term and non-continuous treatment of mild to moderate eczema (atopic dermatitis) in non-immunocompromised people 12 and older whose disease is not well controlled with topical prescription therapies or when those therapies are not recommended. The studies TRuE-V1 and TRuE-V2 assessed Opzelura in more than 600 patients ages 12 and older with non-segmental vitiligo and are currently undergoing FDA (United States Food and Drug Administration) review for the indication of vitiligo. The 52-week results build on the previously announced positive 24-week results and include data from the 24-week double-blind and 28-week treatment extension periods of the Phase 3 TRuE-V1 and TRuE-V2 studies. Findings from the Week 52 analysis showed that patients applying 1.5% ruxolitinib cream twice daily (BID) had clinically meaningful facial and total body repigmentation as shown by greater proportions of patients reaching the facial and total body Vitiligo Area Scoring (F-VASI [facial vitiligo area scoring index] and T-VASI [total body Vitiligo Area Scoring Index], respectively) endpoints at Week 52. Specifically, efficacy results of patients who applied ruxolitinib cream from Day 1 showed that at Week 52, approximately 50% of patients achieved 75% improvement in the facial Vitiligo Area Scoring Index (F-VASI75) compared to the F-VASI75 (75% improvement in the facial Vitiligo Area Scoring Index) improvement from baseline reported for these patients at Week 24 (the primary endpoint of the study) which was approximately 30%. The overall safety profile of ruxolitinib cream in vitiligo was consistent with previous study data there were no clinically significant application site reactions or serious treatment-related adverse events related to ruxolitinib cream.

A recent literature review investigating the use of topical Janus kinase inhibitors (TJKI) for the treatment of vitiligo found that TJKI (topical Janus kinase inhibitors) offer an attractive new treatment option for the management of vitiligo. Preliminary reports suggest that they are more effective than TCI (topical calcineurin inhibitors) and at least equal in efficacy to TC (topical corticosteroids). 

This patient has failed topical steroids and calcineurin inhibitors. It is medically appropriate for him to try ruxolitinib cream, as the published literature to date supports it as a viable treatment option for this diagnosis.