FDA Approves Dupixent as First Targeted Treatment for Bullous Pemphigoid in Adults

Today, the FDA approved Dupixent (dupilumab) as the first and only targeted therapy for adult patients with bullous pemphigoid (BP), a rare, chronic and painful inflammatory skin disease.

This approval marks the eighth disease indication for Dupixent as a new alternative for patients—many of whom are elderly and have limited treatment options.

BP is an autoimmune skin disorder that mainly affects older adults and causes significant suffering due to severe itching, blisters, lesions and skin breakdown.

The approval was based on results from the ADEPT Phase 2/3 trial, where Dupixent significantly improved disease control, reduced itching and lowered reliance on oral corticosteroids (OCS), which are currently the standard treatment but can carry serious side effects.

Dupixent is a fully human monoclonal antibody that works by inhibiting the signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13)—key drivers of type 2 inflammation, which plays a crucial part in several chronic allergic and inflammatory conditions.

BP is an autoimmune skin disorder that mainly affects older adults and causes significant suffering due to severe itching, blisters, lesions and skin breakdown.

These symptoms can make daily activities difficult and increase a person’s risk of infection.

Treatments have traditionally relied on systemic immunosuppressants that carry a range of safety concerns, especially in the elderly population. However, Dupixent is not an immunosuppressant.

It was found in a 2018 study published in Frontiers in Medicine that the estimated global incidence of BP ranges from 2.4 to 21.7 new cases per million people annually.

In the U.S., a 2019 study in the Journal of the American Academy of Dermatology found a prevalence of 12 per 100,000 adults, which increases to nearly 38 per 100,000 among those aged 60 and older.

For BP, Dupixent is administered through subcutaneous injection at a dose of 300 mg every other week after a loading dose, in combination with a tapering course of oral corticosteroids.

While the drug is typically given in a clinical setting, patients can administer the injection at home after proper training from a healthcare provider, according to a news release by Sanofi.

Drugs.com shared the general listing price for Dupixent at approximately $4,273 for a 4 mL supply at the 300 mg/2 mL concentration.

Based on the ADEPT Phase 2/3 clinical trial—which evaluated Dupixent in 106 adults with moderate-to-severe BP—participants were randomized to receive either Dupixent or a placebo in addition to standard oral corticosteroids and underwent a defined tapering protocol for steroid use.

Results revealed that by week 36, 18.3% of patients on Dupixent achieved sustained disease remission, compared to 6.1% in the placebo group.

Itch reduction was seen in 38.3% of Dupixent patients versus 10.5% on placebo. The median cumulative OCS dose was 2.8 grams with Dupixent and 4.1 grams with placebo.

Side effects observed in at least 2% of patients taking Dupixent included joint pain (arthralgia), conjunctivitis, blurred vision, herpes viral infections and keratitis.

One patient on Dupixent experienced a case of acute generalized exanthematous pustulosis, which did not occur in the placebo group.

George D. Yancopoulos, M.D., Ph.D., board co-chair, president, and chief scientific officer at Regeneron, said in the news release that the approval extends “the remarkable ability of Dupixent to transform treatment paradigms for people living with a variety of diseases with underlying type 2 inflammation.”

He added that the drug’s approval “further reinforces the demonstrated safety profile of Dupixent in a broad age range of patients, from infants to elderly people and across dermatological, respiratory and gastrointestinal diseases.”

The FDA evaluated the application under priority review. Dupixent also received orphan drug designation, according to the release.

Additionally, regulatory submissions for Dupixent in BP are also under review in Europe, Japan and China.

Dupixent is approved in more than 60 countries for one or more indications, including atopic dermatitis, asthma, eosinophilic esophagitis and prurigo nodularis, and is being used by over one million patients worldwide.

The drug is jointly developed by Sanofi and Regeneron, with an ongoing research program spanning over 60 clinical trials and more than 10,000 patients.

Future potential uses—such as for chronic pruritus of unknown origin and lichen simplex chronicus—are still under investigation.