Spesolimab (SPEVIGO®), a humanized anti-IL-36 IgG1k antibody developed by Boehringer Ingelheim, was approved by the FDA as a treatment option for generalized pustular psoriasis (GPP) flares in adults, as announced by BI on September 1, 2022. GPP is a rare and potentially life-threatening neutrophilic skin disease characterized by episodes of widespread eruptions of painful, sterile pustules. The FDA had previously granted spesolimab Breakthrough Therapy and Orphan Drug designations for the treatment of GPP, and the BLA for spesolimab received a Priority review. In addition, spesolimab has received Breakthrough Therapy Designation in China and Taiwan, Priority Review in the China, Orphan Drug Designation in Korea, Switzerland and Australia, and Rare Disease designation and fast track in Taiwan for the treatment of GPP flares. An MAA for use of spesolimab as a treatment of flares in GPP is undergoing evaluation by the EMA.
The approval by FDA was based in part on results from the 12-week pivotal Phase 2 Effisayil™ 1 clinical trial (NCT03782792), which evaluated the efficacy, safety, and tolerability of a single 900 mg dose of IV administered spesolimab, with the option of a second dose if symptoms persisted on Day 8, vs placebo in 53 patients experiencing a GPP flare. After one week, 54% of patients treated with SPEVIGO showed no visible pustules compared to 6% of those who received placebo. A 3-arm, 5-year Phase 2 study (NCT03886246) to evaluate spesolimab in GPP patients who took part in previous studies with spesolimab is currently recruiting an estimated 155 participants. Patients will be administered SPEVIGO® at 4-, 6- or 12-week intervals. The primary outcome measure of the study is the occurrence of treatment emergent adverse events (TEAEs) up to week 252 of maintenance treatment; secondary outcome measures relate to the efficacy of the drug.