The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to spesolimab for the prevention of flares in adults with generalized pustular psoriasis (GPP), a potentially life-threatening chronic neutrophilic skin disease.
Spesolimab is a humanized monoclonal immunoglobulin G1 antibody that inhibits interleukin-36 (IL-36) signaling. It is currently approved as an intravenous infusion therapy under the brand name Spevigo for the for the treatment of GPP flares in adults.
The Breakthrough Therapy designation was based on data from the double-blind, placebo-controlled phase 2b Effisayil 2 trial (ClinicalTrials.gov Identifier: NCT04399837), which evaluated the safety and efficacy of maintenance treatment with subcutaneous spesolimab for the prevention of GPP flares in patients with a history of GPP.
Findings showed that spesolimab met the primary endpoint, demonstrating significant prevention of GPP flares for up to 48 weeks. The safety profile of spesolimab was found to be consistent with what has been previously observed in other clinical trials.
“GPP flares can appear suddenly and intensify quickly, often leaving patients with a heavy emotional burden,” said Claudia Beqaj, Executive Director, Dermatology, Sales and Marketing, Boehringer Ingelheim. “The FDA’s action reinforces the potential of spesolimab and represents a critical step in bringing this treatment to patients who need it most. We look forward to working with the FDA to accelerate the development of this treatment.”
US FDA grants Breakthrough Therapy designation for spesolimab for the prevention of flares in adults with generalized pustular psoriasis (GPP). News release. Boehringer Ingelheim. May 2, 2023. https://www.prnewswire.com/news-releases/us-fda-grants-breakthrough-therapy-designation-for-spesolimab-for-the-prevention-of-flares-in-adults-with-generalized-pustular-psoriasis-gpp-301813047.html.