Finding the right treatment for vitiligo — a condition that causes patches of skin to lose color — can be challenging. Researchers have been working on new options to help make vitiligo treatment faster and more effective. One experimental treatment, afamelanotide, may help people with vitiligo get better, faster results. Keep reading to learn more about afamelanotide and how it may help treat vitiligo.

What Is Afamelanotide?

Afamelanotide is a drug that helps skin cells make eumelanin — a type of melanin, the natural pigment that gives color to your skin, eyes, and hair. Sold under the brand name Scenesse, afamelanotide is given as a small implant (a device placed under the skin near the hip). The U.S. Food and Drug Administration (FDA) has approved afamelanotide to treat erythropoietic protoporphyria (EPP). This rare condition causes skin pain and swelling after sun exposure.

Although afamelanotide is approved only for EPP, researchers have also studied it to see if it can treat vitiligo and other conditions, such as:

• Solar urticaria, which causes itchy rashes after sun exposure
• Polymorphic light eruption, also called sun allergy, in which rashes occur after sun exposure in spring and early summer
• Hailey-Hailey disease, a painful rash that often appears in skin folds like the armpits or groin

How Can Afamelanotide Help Vitiligo?

Vitiligo is an autoimmune disease, which means the immune system mistakenly attacks the body’s own cells. In vitiligo, the immune system attacks and destroys melanocytes (skin cells that make melanin). When an area of skin loses melanocytes, it has less melanin, leading to white patches.

Researchers began studying afamelanotide for vitiligo because of the drug’s potential to make more eumelanin. Afamelanotide is an analog (lab-made version) of a natural hormone called alpha-melanocyte-stimulating hormone (alpha-MSH). This hormone normally binds to a receptor on skin cells called melanocortin-1 receptor (MC1R). When alpha-MSH binds to MC1R, it signals the skin to make more eumelanin. Because afamelanotide looks like alpha-MSH, it can also help the skin make more color.

In vitiligo treatment, afamelanotide is combined with a type of phototherapy called narrow-band ultraviolet B (NB-UVB). NB-UVB phototherapy is often recommended as the first treatment for people with generalized (full-body) vitiligo. On its own, NB-UVB phototherapy works slowly over time. However, when it’s combined with afamelanotide, skin pigmentation may happen faster.

The two treatments work well together because NB-UVB stimulates skin cells to make more MC1R. With more of these receptors present, afamelanotide can be even more effective at stimulating the skin to make more eumelanin.

How Effective Is Afamelanotide for Vitiligo?

Afamelanotide is still being studied as a vitiligo treatment in U.S. and European clinical trials (studies that look at a new therapy’s safety and effectiveness). So far, only a few small studies have looked at how well afamelanotide helps with repigmentation.

The first report on afamelanotide for vitiligo, published in 2013, included four participants with generalized vitiligo. Each person received an implant along with NB-UVB phototherapy. All four people began to see repigmentation within one to four weeks. These early results helped researchers see that afamelanotide might be a useful treatment for vitiligo.

In 2015, a larger clinical trial that also included those four participants was published. This study involved 55 participants from several dermatology clinics in the U.S. The participants were split into two groups. One group received afamelanotide plus NB-UVB phototherapy, and the other (the control group) received only NB-UVB. After six months, the afamelanotide group had faster and better skin repigmentation compared to the control group. The researchers also found that afamelanotide worked especially well in people with darker skin tones.

Another small study published in 2020 showed that afamelanotide combined with NB-UVB phototherapy was more effective than NB-UVB alone in Asian people with vitiligo.

More clinical trials are underway to better understand how afamelanotide can help treat vitiligo.

How Do You Use Afamelanotide?

Afamelanotide is given as a small implant — about the size of a grain of rice — that goes under the skin. A trained healthcare provider inserts the device, usually near the hip bone. Before placing the implant, your healthcare provider will numb the area so that you don’t feel pain. After the implant is inserted, you’ll stay at the clinic for at least 30 minutes to make sure you don’t have an allergic reaction.

The implant usually doesn’t need to be removed because it slowly dissolves over time.

How Often Do You Get an Afamelanotide Implant for Vitiligo?

Afamelanotide is being studied as combination therapy with NB-UVB phototherapy. Because afamelanotide is only approved to treat EPP, it’s considered an off-label (not officially approved) treatment for vitiligo. People with EPP get a new implant every two months, usually receiving three or four implants per year.

Afamelanotide was given more often in clinical trials for vitiligo. In the 2015 study, participants received a new afamelanotide implant every month for four months. They also had NB-UVB phototherapy two or three times a week for six months.

If you start afamelanotide therapy for vitiligo, your healthcare team may suggest a different treatment schedule.

What Are the Risks of Afamelanotide for Vitiligo?

Afamelanotide research has mainly focused on people with EPP. In those studies, most people tolerated the treatment well and didn’t have any serious side effects. When side effects did happen, they usually occurred in the first few days after the implant was inserted.

Common side effects of afamelanotide in people with EPP include:

• Discoloration or irritation at the implant site
• Nausea
• Mouth or throat pain
• Cough
• Fatigue (tiredness that doesn’t improve with rest)
• Dizziness
• Drowsiness
• Respiratory tract infection, such as a cold or flu
• Skin irritation
• Nonacute porphyria (a condition in which certain substances build up in the body instead of being used to make hemoglobin, the protein that carries oxygen in blood)

Afamelanotide may also cause changes in skin color. The treatment can cause tanning of normal skin, making some areas look darker than others. Moles or birthmarks also may get darker during treatment. Because of this, researchers and doctors recommend having a full skin examination twice a year while using afamelanotide to monitor for skin changes.

More clinical trials are needed to help researchers better understand the side effects of afamelanotide specifically in people with vitiligo. In the 2015 study, the side effects were similar to those of people with EPP. In vitiligo treatment, there may also be side effects from NB-UVB phototherapy, including discolored, dry, rough, or scaly skin.

The good news is that neither afamelanotide nor NB-UVB phototherapy has been shown to increase the risk of skin cancer, such as melanoma. However, it’s important to talk to your doctor about your skin cancer risk.

How Can You Access Afamelanotide Treatment?

Afamelanotide isn’t FDA-approved to treat vitiligo at this time. This therapy is still being tested in clinical trials for people with vitiligo.

The studies completed so far have given researchers enough information to continue with larger clinical trials. These larger studies, with more people who have vitiligo, will help researchers understand if afamelanotide works differently for vitiligo than it does for EPP, as well as whether the side effects are similar or different.

If you’re interested in afamelanotide treatment for vitiligo, talk to your dermatologist. They can help you find out whether you might qualify for a clinical trial and explain what’s involved.

Talk With Others Who Understand

MyVitiligoTeam is the social network and support group for people with vitiligo and their loved ones. On MyVitiligoTeam, more than 14,000 members come together to ask questions, give advice, and share their stories with others who understand life with vitiligo.

Have you participated in a clinical trial involving afamelanotide? Are you curious about how this treatment might work for vitiligo in the future? Share your experiences in the comments below, or start a conversation by posting on your Activities page.

In partnership with the Global Vitiligo Foundation, which strives to improve the quality of life for individuals with vitiligo through education, research, clinical care, and community support.