Sanofi and Regeneron Aim To Win Approval For Dupixent In Bullous Pemphigoid After Promising Phase 3 Results

Sanofi and Regeneron’s acclaimed anti-inflammatory biologic, Dupixent, is continuing its impressive streak. On the same day the companies announced encouraging results from a study on chronic spontaneous urticaria (CSU) and a fresh analysis of data in chronic obstructive pulmonary disease (COPD), new findings from a key study in bullous pemphigoid (BP) highlight the drug's potential for another regulatory submission. In the ADEPT study, involving 106 BP patients, those receiving 300 mg of Dupixent every two weeks achieved sustained disease remission at a rate of 20%, which is five times higher compared to the 4% remission rate observed in the placebo group, as reported by Sanofi on Wednesday.

The goal was defined as achieving full clinical remission after patients had stopped taking standard oral corticosteroids (OCS) by the 16th week, with no occurrences of relapse or need for additional treatments during the 36-week trial. Sanofi also reported that Dupixent outperformed placebo on several secondary measures, including a decrease in disease severity and significant improvement in itch, among other factors. 

This chronic, recurring condition is characterized by reddened skin, persistent painful lesions, severe itching, and blistering that can spread across large areas of the body, making patients susceptible to infections. It predominantly affects individuals aged 60 to 80. Sanofi’s chief medical officer and global head of development, Dietmar Berger, M.D., Ph.D., emphasized in the release that the blisters associated with the disease can be "debilitating," particularly for older patients.

Berger said, “There is a significant unmet medical need for new medicines for people suffering with this hard-to-treat disease in which the standard of care is oral and topical corticosteroids and immunosuppressants — treatments that have poor clinical outcomes and safety concerns, respectively, and should be used sparingly in an elderly population. Combined with the consistent safety profile of the other dermatology indications, these results show the potential of Dupixent to transform the treatment paradigm for bullous pemphigoid.”

In the older demographic studied, the frequency of adverse events was consistent between those treated with Dupixent and those given a placebo, both at 96%. However, certain adverse effects, such as peripheral edema, blurred vision, and asthma, were more prevalent among Dupixent recipients. The release noted that no adverse events resulted in death in the Dupixent group, whereas two deaths occurred in the placebo group. In addition to the successful ADEPT trial, a separate phase 3 study assessed Dupixent's efficacy in adults with severe chronic pruritus of unknown origin (CPUO). Despite showing some positive numerical improvements, this trial did not achieve its primary endpoint, according to Sanofi.

Dupixent, which was earlier granted orphan drug status by the FDA for Bullous Pemphigoid (BP), is now set to leverage its recent positive study results to seek global regulatory approvals, starting with the FDA. If it secures approval, Dupixent would become the first targeted therapy for BP in both the U.S. and Europe, according to Sanofi.

The positive BP study results were announced alongside Sanofi's report of promising findings in Chronic Spontaneous Urticaria (CSU), following an earlier FDA rejection for that indication. Sanofi plans to pursue a label expansion for CSU based on these results. Additionally, Sanofi highlighted that a combined analysis of two Phase 3 trials has confirmed Dupixent's significant benefits in treating COPD. The FDA is expected to make a decision on Dupixent's COPD approval by September 27, following a prior delay.