Oplezura (ruxolitinib) is currently the first and only approved treatment in the UK for non-segmental vitiligo that supports repigmentation.

Oplezura, manufactured by Incyte Corporation, is a topical cream with the active component ruxolitinib used for vitiligo treatment (1). It was approved by the UK’s Medicine and Healthcare Products Regulatory Agency in July 2023 for treatment of non-segmental vitiligo with facial involvement in adults and children over 12 years old to aid repigmentation (2). The treatment is currently available on private prescription to eligible patients, however, as of the latest National Institute for Health and Care Excellence (NICE) update, a decision on the treatment’s availability on the NHS is expected on 31 st July 2024 (3). This article aims to provide you with the overview on how Oplezura (ruxolitinib) works, its effectiveness based on the recent clinical trials, the recommended use of the cream and its side effects.

Oplezura is a prescription-only steroid-free cream applied to the skin (4). It is sold in either 60 g or 100 g tubes. The active substance in the medication is ruxolitinib (ruxolitinib phosphate; 5). It is a 1.5% ruxolitinib cream, meaning that each gram of Oplezura contains 15 mg of the active compound. Besides repigmentation support in vitiligo, the cream is also used for treatment of mild and moderate eczema (6). In the UK, ruxolitinib is also licensed in a tablet form (Jakavi) for treatment of certain blood disorders, such as myelofibrosis or polycythaemia vera (7).

Ruxolitinib is one of the Janus kinase (JAK) inhibitors used to treat chronic inflammatory disorders. JAK1 and 2 are enzymes that play a key role in the immune pathway responsible for melanocyte (pigment cell) destruction in vitiligo. Ruxolitinib blocks those enzymes and reduces the immune system’s ability to destroy melanocytes (8). Specifically, ruxolitinib has three effects on vitiligo, as explained by Incyte (9):

Repigmentation happens by reducing the autoimmune activity in the skin, which then encourages the melanocytes to travel from vitiligo lesion edges and hair follicles into the lesion itself and produce pigment (10).

In 2022, the results of two big Oplezura trials, TRuE-V1 and TRuE-V2, were combined and published as one study (11). The study investigated the effectiveness of Oplezura on patients with vitiligo. It had over 650 participants from North America and Europe, each over 12 years of age with non-segmental vitiligo, with depigmented areas covering 10% or less of total body surface area, including both the face and the rest of the body. The researchers monitored patients for up to 2 years for changes in their vitiligo lesions.

Most participants found marked improvements with ruxolitinib, particularly when it came to vitiligo patches on the face. However, there was some variation in how quickly participants responded to the treatment. Roughly one third of the participants achieved a 75% repigmentation of the patches on their face at 6 months of treatment. Half the patients achieved that in a year. Two thirds of patients saw the same improvement at 2 years (although some participants dropped out of the study). Some participants – 15% – saw as much as 90% repigmentation of the lesions on their face at 6 months.

Oplezura was slightly less effective on non-facial areas of the body with around a quarter of the participants achieving 50% repigmentation of the lesions on their whole-body surface area after 6 months.

For a more detailed, patient-friendly summary of the results of TRuE-V1 and TRuE- V2 please visit Incyte’s Oplezura website.

While the results of the study appear very promising, it is important to remember that repigmentation takes time. The pigment cells, melanocytes, destroyed in vitiligo have to regenerate and migrate from healthy skin to the lesions. This is a slow healing process and will not happen overnight – just like when you scrape your knee, your body may take quite a while to completely heal the injury. Some parts of the body will repigment faster than others and some may not respond to the treatment at all. Areas with completely white hairs are unlikely to repigment. There are many patterns in which a lesion may repigment and your healthcare provider should teach you how to look for signs of this (4, 10). Tracking your progress with photos may help as well. The next section will explain how to make sure you are benefitting the most from the treatment.

Oplezura is a prescription-only cream that can be applied anywhere on the body, including sensitive areas like around the eyes, mouth and genitalia. It is important to make sure the cream does not enter the eyes or mouth as it is intended for skin use only. Apply the cream twice daily with a minimum of 8 hours between the two applications – ideally as part of the morning and evening routines. A thin layer should be spread on the smallest area of the skin necessary. You may apply the cream on up to 10% of the body surface area affected by vitiligo which equals to about the size of ten handprints. Avoid washing treated skin for at least 2 hours after application. You should wash your hands after applying the cream (unless the skin there is being treated). To maximize the effectiveness of Oplezura, make sure to use it as intended – twice a day – otherwise you may not make the most of the treatment. You should use up to one 60 g tube of the cream per week, or up to one 100 g tube over two weeks. Oplezura should be stored at room temperature.

You should track your progress with regular photos. After prescribing Oplezura, the doctor will schedule follow-up appointments to monitor your progress and to examine your skin. If you achieve less than 25% repigmentation after a year of treatment, the doctor may decide that the treatment is not appropriate for you and stop it.

Oplezura is not licensed for use in pregnancy. Patients should not breastfeed during treatment and for about four weeks after the last dose. People of childbearing potential have to use effective contraception during treatment and for four weeks after the treatment is stopped.

Oplezura should not be used in combination with therapeutic biologics, other JAK inhibitors or potent immunosuppressants (such as azathioprine or cyclosporine). It is unlikely to interact with other drugs, such as medications for high blood pressure or antidepressants, since it is a topical drug. Patients must inform their healthcare provider if they have ever had any type of cancer before receiving the prescription.

You are very unlikely to experience serious side effects when using Oplezura, as it is a topical cream only. Research has found that the most common side effect of the medication is application site acne, experienced by up to 10% of patients. Another common side effects were itching and redness of the application side, as well as headaches, runny nose and fevers. As ruxolitinib is a JAK inhibitor you may be at increased risk for developing infections, such as urinary tract infections. Studies have found a very small increase in the risk of developing skin cancer, as well as cardiovascular events such as heart attacks or blood clots, particularly if you have
had any of those in the past. It is important to inform your healthcare provider if that happens to be the case. You will see your healthcare provider regularly for skin inspections and other health monitoring to minimise those risks.

NICE had their second committee meeting about ruxolitinib on the 6th of June 2024 (3). Currently, the decision on whether ruxolitinib should be available on the NHS for eligible patients is expected on 31 st July 2024. NICE base their decisions on the effectiveness of a treatment, the balance between the benefits and potential harms, as well as the quality of available evidence (12). The Vitiligo Society will keep you updated regarding any decisions from NICE on the matter. Ruxolitinib is a very promising treatment for vitiligo and making the treatment accessible on the NHS could make a huge difference for those affected by it.

References

Funding disclosure:
The Vitiligo Society has project work supported by an educational grant from Incyte Biosciences UK Ltd. The Vitiligo Society maintains full control over the all project management and content, ensuring independence and impartiality in its work.

When it comes to any external funding we follow the current guidance and rules as set out by the Charity Commission and the Fundraising Regulator. We report any research collaborations and/or financial contributions received from industry in our annual reports and accounts as well as being transparent about our partnerships on our website.