The U.S. Food and Drug Administration has finally approved a first-of-its-kind treatment for vitiligo by Incyte. 

DRUG 

Opzelura (1,5% ruxolitinib cream) is the only one* drug approved in the U.S. to restore skin color in people with the most common, non-segmental form of vitiligo. The medication is approved for topical use in ages 12+, twice daily to affected areas of up to 10% body surface area, with no limits on usage duration or areas of application**, — as long as patients continue to see benefit. 

Incyte noted it could take up to 24 weeks for Opzelura to work for some vitiligo patients, yet satisfactory outcomes may require treatment for up to one year. Authors of a recently published meta-analysis paper suggest that "concurrent UVB phototherapy appears to improve efficacy of JAK inhibitors for vitiligo" although it remains unclear whether patients on Opzelura will observe any change following phototherapy.

In its research for new drugs, Incyte researchers discovered that ruxolitinib (wiki) was effective in treating autoimmune diseases, including vitiligo and atopic dermatitis (eczema). After a multi-year clinical study of topical form of ruxolitinib, the results for vitiligo were remarkably effective. It was shown to help about 30% of patients regain more than 75% of their natural pigmentation in the facial area by the six-month mark. Improvements grew to 50% of patients by the one-year mark and the cream proved easy to apply and well-tolerated, with few adverse reactions to the medication. 

Last year, ruxolitinib won FDA approval for Opzelura for eczema. In December, the FDA accepted Incyte’s application for Opzelura and agreed to give it an expedited review. A verdict was expected by April — but in mid-March, Incyte disclosed the agency needed more time to conduct its review and set a new decision deadline of July 18, which was honored by the FDA this time.

PATIENTS 

Incyte estimates up to 3 million people in the U.S. have vitiligo. Of those, about 1.5 million have an official diagnosis and upwards 200,000 are actively seeking treatment. Another active participant of the vitiligo drug race, Pfizer has conducted it's own research to conclude that the current prevalence estimate of vitiligo in the U.S. adults is between 0.76% (1.9 million cases) and 1.11% (2.8 million cases).

In preparation for the commercial rollout of Opzelura for vitiligo, Incyte has reportedly signed contracts with three largest pharmacy benefit managers and group purchasing organizations that cover 80% of the patients in the USA. All three have a general policy that they will automatically cover any FDA-approved drug that costs $100,000 or less per quality-adjusted life years.

It can be challenging to estimate how much the drug will cost to a patient, as all depends on the individual's insurance plan. However, Incyte intends to "provide co-pay cards that will limit the out-of-pocket cost to patients to hopefully as little as $10 or less," according to Dr. Stein, Chief Medical Officer at Incyte. Company also offers IncyteCARES Patient Assistance Program for those without prescription coverage and gross family household income at or less than $129,800.

Despite having first-mover status, activating the vitiligo patient community won’t be easy. It has grown frustrated by the frequent misdiagnosis, lack of effective therapies, side-effects from improper use of off-label treatments, insurance coverage hurdles, and so on. Recently, the company has launched the Incyte Ingenuity Awards in vitiligo with $135K funding of two innovative initiatives that address challenges faced by patients, caregivers, and healthcare providers.

BUSINESS 

"With what we believe are relatively conservative assumptions for about 25,000 patients treated and the company's reiterated expectation for an average of 10 tubes per year (per patient)…”, said RBC analyst Brian Abrahams. On the news, Incyte’s share price was up 2%, to around $80.50, in late Tuesday morning trading.

The FDA approval of Opzelura for vitiligo will start a full-scale commercial roll-out and marketing campaign within a few months, but Incyte is also eyeing European approval for vitiligo treatment for as early as the end of this year.

SAFETY

Opzelura (Ruxolitinib) belongs to a class of drugs known as Janus kinase inhibitors, or JAK inhibitors. JAK inhibitors are anti-inflammatory therapies that combat diseases at the immune system level. They target the JAK-STAT pathway, a signal mediator for cytokines associated with several skin diseases, like psoriasis, itch, atopic dermatitis, alopecia areata and vitiligo. JAKs work incredibly quickly—on the order of hours to a few days. Yet, while providing health benefits, JAK inhibitors can lower the ability of the immune system to fight certain infections. 

In recent years, drug agencies have taken issue with the safety of JAK inhibitors developed by Pfizer, Eli Lilly and others. Xeljanz, Olumiant and Rinvoq in this class can increase the risk of cardiovascular-related events, cancer, blood clots and death. 

But that doesn't appear to be the case for Opzelura. The FDA has updated the black box and safety warnings to more specifically distinguish serious adverse events related to topical JAK inhibitors like Opzelura from the general oral kind. 

The most common side effects of Opzelura in people treated for vitiligo include: acne, itching or redness at the application site, headache, common cold (nasopharyngitis), fever, and urinary tract infection. Patients may be at a higher risk of developing shingles (herpes zoster) while using the medication.

Use of Opzelura in combination with therapeutic biologics, other JAK inhibitors, or potent immunosuppressants, such as azathioprine or cyclosporine, is not recommended.

It is not known if Opzelura is safe and effective in children less than 12 years of age with atopic dermatitis or non-segmental vitiligo.

COMMUNICATION

Incyte will host an analyst and investor conference call and webcast on July 19, 2022 at 8:00 a.m. EDT. The live and archived webcast will be available via Investor.incyte.com. The live webcast with slides can be accessed at Investor.Incyte.com and will be available for replay for ninety days.

N.B.

* Technically, Opzelura is not the first FDA-approved drug for vitiligo. Monobenzyl ether of hydroquinone (MBEH) was approved by FDA under trade name Benoquin well before Opzelura. It is also known as “depigmenting,” “whitening” or “bleaching” cream. When a few islands of natural skin color vividly stand out against a sea of milky white vitiligo, depigmentation is often the only option to create a flawless look.

While MBEH is intended to remove the remaining skin color, Opzelura is intended to restore the original skin color. Thus, it fully deserves the “first-in-class” title for vitiligo treatment. 

** except for eyelids, mouth and vagina