Withdrawal of EMA application for the use of Dupixent (dupilumab) in the treatment of adults with moderate-to-severe bullous pemphigoid – Sanofi

Sanofi withdrew its application for the use of Dupixent (dupilumab) in the treatment of adults with moderate-to-severe bullous pemphigoid, an autoimmune skin disease causing widespread itching and blisters affecting the skin and sometimes the inside of the mouth. Dupixent has been authorised in the EU since September 2017. It contains the active substance dupilumab and is available as pre-filled pens or syringes containing dupilumab in a solution for injection under the skin. The company applied for an extension of indication to add the treatment of bullous pemphigoid in adults. During the procedure, the company amended the indication to the treatment of moderate to severe bullous pemphigoid in adults.

The company presented the results of a main study involving 106 adults with bullous pemphigoid. Patients received either Dupixent or placebo in addition to background treatment of oral corticosteroid medicines until patients achieved stable disease control. The main measure of effectiveness was the proportion of patients who achieved sustained remission, defined as no need for corticosteroids by week 16 of treatment and no disease relapse (when symptoms come back) and need for rescue therapy by week 36 of treatment.

The application was withdrawn after the European Medicines Agency had evaluated the information from the company and had prepared questions for the company. After the Agency had assessed the company’s responses to the questions, there were still some unresolved issues. The main study did not achieve its main objective and the results for the other outcomes were not robust enough due to limitations with the study design. Any improvements seen were modest, with a high level of uncertainty, and the additional supporting information, mainly from real‑world experience, was not sufficient to confirm that the medicine is effective.

Therefore, at the time of the withdrawal, the Agency’s opinion was that the benefits of Dupixent in the treatment of bullous pemphigoid did not outweigh its risks.

In its letter notifying the Agency of the withdrawal of application, the company stated that it withdrew the application due to a divergence of views with the Agency’s scientific committee, the CHMP, regarding the sufficiency of the data supporting the proposed extension of use for Dupixent.

The withdrawal does not impact the authorised uses of this medicine.