
The US Food and Drug Administration (FDA) has granted approval to Dupixent (dupilumab), developed by Sanofi and Regeneron, as the first targeted medication for adults suffering from bullous pemphigoid (BP).
This skin condition primarily impacts the elderly, manifesting in severe itching, painful blisters, and lesions, and is linked to type 2 inflammation. The disease can be chronic and lead to significant infections due to skin breakdown, complicating daily life. Current treatment options are limited and may compromise the immune system.
Sanofi immunology and oncology development global therapeutic area head Alyssa Johnsen said: “Until now, treating bullous pemphigoid was very challenging for elderly patients struggling with the debilitating impact of blisters and lesions, and potentially co-morbid conditions.
“By addressing two central drivers of the underlying type 2 inflammation that contributes to bullous pemphigoid, Dupixent is the first targeted medicine to allow patients the potential to achieve sustained remission and reduce itch.”
The FDA’s decision follows data from the ADEPT Phase 2/3 study assessing Dupixent’s safety and efficacy against a placebo in adults with moderate-to-severe BP.
Participants were divided into groups receiving either Dupixent 300 mg or a placebo alongside standard oral corticosteroids (OCS). A structured OCS tapering was implemented for those showing controlled disease activity.
Final results indicated that 18.3% of patients on Dupixent achieved sustained remission compared to 6.1% on placebo, while 38.3% noted significant relief from itching versus 10.5% on placebo. Moreover, the median cumulative OCS dosage for Dupixent patients was lower at 2.8g compared to 4.1g for the placebo group.
During this study, adverse events such as joint pain, conjunctivitis, blurred vision, herpes viral infections, and keratitis were more prevalent in patients receiving Dupixent. There was one incident of acute generalised exanthematous pustulosis reported among Dupixent users, with none in the placebo group.
Regeneron board co-chair, president, and chief scientific officer George Yancopoulos said: “Dupixent has shown the potential to improve the most challenging effects of bullous pemphigoid, while helping some patients achieve sustained disease remission and decreased oral corticosteroid use.
“Additionally, this 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 performed a priority review of Dupixent due to its potential to significantly enhance treatment efficacy or safety for serious conditions. Previously, it had been given orphan drug status by the FDA for BP treatment due to its role in addressing rare diseases affecting fewer than 200,000 individuals in the US.
Beyond the US, regulatory reviews are underway in regions including Europe, Japan, and China.
Dupixent is administered through subcutaneous injection at varying sites and requires a healthcare professional’s supervision but can also be given at home after proper training. The drug is a monoclonal antibody that targets interleukin-4 (IL4) and interleukin-13 (IL13) pathways without suppressing the immune system.
Globally approved across over 60 countries for different conditions, Dupixent addresses ailments such as atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, prurigo nodularis, chronic spontaneous urticaria, chronic obstructive pulmonary disease (COPD), and now BP across various age groups.