Can Dupixent plug treatment gap in bullous pemphigoid?

Sanofi and Regeneron's Dupixent already boasts a long list of FDA-approved indications, and the latest to be reviewed could throw a lifeline to patients with a rare and debilitating autoimmune disease affecting the skin.

Bullous pemphigoid (BP), which has no FDA-approved targeted therapies, leads to reddening of the skin and the formation of painful and intensely itchy chronic lesions and blisters, in some cases spreading across a majority of the body. It most commonly affects older people in the 60 to 80 age bracket.

IL-4 and IL-13 inhibitor Dupixent (dupilumab) has started a priority review at the FDA and is the first biologic drug to show an improvement in remission and symptoms in BP, and the first to allow patients to reduce the use of steroids, which are the go-to therapy for the disorder but can have serious long-term side effects. The US regulator is due to deliver a verdict on the drug by 20th June.

Sanofi and Regeneron filed Dupixent for BP on the back of the ADEPT study reported last year, which showed that the drug achieved a five-fold greater sustained remission rate at 36 weeks – 20% versus 4% – compared to placebo. Reaching that endpoint meant that skin lesions had been resolved, with no relapse, and standard treatment with oral corticosteroids had been tailed off without the need for any rescue therapy.

In addition, 41% of Dupixent-treated patients achieved a 90% or greater reduction in disease severity, compared to 10% of the control group, while "clinically meaningful" itch reduction was recorded in 40% and 11%, respectively.

The International Pemphigus & Pemphigoid Foundation (IPPF) has hailed the results as a landmark for the BP patient community, whose needs "have gone unmet for far too long," according to executive director Patrick Dunn.

There are around 27,000 adults in the US who live with BP that cannot be controlled by systemic corticosteroids. Currently, their treatment options are largely limited to powerful immunosuppressant therapies that also carry a significant side-effect burden.

BP is one of two new indications that could be on the way for Dupixent this year, which is already a $13.6 billion blockbuster from approved uses in diseases like atopic dermatitis and eosinophilic asthma. The other is chronic spontaneous urticaria (CSU), for which Dupixent was turned down in 2023 but refiled last year with an FDA action date of 18th April.