Sanofi, Regeneron to seek approval for Dupixent in bullous pemphigoid after positive phase 3 study

Sanofi and Regeneron’s superstar anti-inflammatory biologic Dupixent is on a roll. On the same day the companies unveiled positive data from a study in chronic spontaneous urticaria (CSU) and a new analysis of results in chronic obstructive pulmonary disease (COPD), results from a pivotal study in rare skin condition bullous pemphigoid (BP) stand to support another upcoming regulatory filing. 

In the 106-patient ADEPT study, BP patients treated with 300 mg of Dupixent every two weeks achieved sustained disease remission at a rate five times higher (20%) than the 4% rate recorded for the placebo group, according to a Sanofi press release on Wednesday

The measure was defined by complete clinical remission after patients tapered off of standard-of-care oral corticosteroids (OCS) by week 16, with no relapses or rescue therapy use over the 36-week trial period.

Dupixent also bested placebo on secondary endpoints such as a reduction in disease severity and clinically meaningful itch, among other measures, Sanofi said.

The chronic and relapsing disease is hallmarked by skin reddening, painful chronic lesions and intense itch and blisters that can form over much of the body and leave patients prone to infection. It most commonly affects people between the ages of 60 and 80.

The blisters caused by the disease can be “debilitating,” especially for elderly patients, Sanofi’s chief medical officer and global head of development, Dietmar Berger, M.D., Ph.D., noted in the release. 

“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,” Berger said. “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 population that the drug was studied in, overall rates of adverse events were the same at 96% for those on Dupixent and on placebo. However, several adverse events were more common in the Dupixent group, including peripheral edema, blurred vision, asthma and others. No adverse events led to deaths in the Dupixent group while two did in the placebo group, according to the release.

Besides the positive ADEPT trial, a separate phase 3 study looked at the use of Dupixent in adults with related disease uncontrolled and severe chronic pruritus of unknown origin (CPUO). That trial did not meet its primary endpoint “despite favorable numerical improvements," Sanofi said.

Dupixent previously was granted orphan drug designation from the FDA in BP and the companies plan to use the positive study to support bids for approval with regulatory agencies across the world, starting with the FDA. If approved, Dupixent would become the first targeted treatment for BP in the U.S. and Europe, according to Sanofi.

The BP results came the same day that Sanofi touted positive results in CSU following a prior FDA rejection in that use. Like with BP, the partners plan to file for a label expansion into CSU.

Also Wednesday, Sanofi said that a pooled analysis of two phase 3 studies "reinforce" the drug's "clinically meaningful benefit" in COPD. The FDA is set to decide on Dupixent's potential approval in COPD by September 27 after a prior delay.