FDA approves Boehringer rare skin disease mAb

Sept. 6, 2022

Boehringer Ingelheim announced last week that the U.S. FDA approved its monoclonal antibody treatment option, spesolimab, for generalized pustular psoriasis (GPP) flares in adults.

The drug blocks the activation of the interleukin-36 receptor, a signaling pathway that has been implicated in the pathogenesis of GPP. GPP is a rare, neutrophilic skin disease, which is a group of conditions characterized by skin lesions caused by neutrophil accumulation. The condition is different from general psoriasis; if left untreated, the flares can cause life-threatening complications such as multisystem organ failure or sepsis.

According to Boehringer Ingelheim, spesolimab, which will be marketed in the U.S. as Spevigo, is the first approved treatment option for GPP.

Spevigo was approved based on results from the phase 2 clinical trial, Effisayil 1. Effisayil 1 (NCT03782792) evaluated the drug’s performance against placebo and included 53 patients. Boehringer reports that after just one week, half of the patients treated with Spevigo had no visible pustules, while only 6% of patients on placebo had visible results. 

Now Boehringer is seeking approval from other international regulatory authorities. The drug has received Breakthrough Therapy Designation in the U.S., China, and Taiwan, Priority review in the U.S. and China, and Orphan Drug Designation in many other countries.