FDA Grants Breakthrough Therapy Designation For Spesolimab

The US Food and Drug Administration (FDA) granted a Breakthrough Therapy designation for spesolimab (Spevigo; Boehringer Ingelheim, Inc.). Previous clinical trials indicated the injection can prevent flares in adults with generalized pustular psoriasis (GPP). The FDA’s designation will allow for an expedited process to develop and review spesolimab to treat patients with this life-altering diagnosis.¹

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Mark Lebwohl, MD has been practicing dermatology since 1983 and currently serves as the dean for clinical therapeutics at the Icahn School of Medicine at Mount Sinai and professor and chairman emeritus of the Kimberly and Eric J. Waldman Department of Dermatology. He is well versed in GPP treatment and was optimistic about today's announcement.

"Spesolimab has been a lifesaving treatment for generalized pustular psoriasis which is a devastating disease. Patients who are critically ill, covered in pustules, and on the verge of death can be restored to normal within hours to days," Lebwohl said. "By providing breathrough therapy designation for spesolimab as a treatment to prevent flares of GPP, there is hope that we will soon have available a therapy to prevent life-threatening flares that plague patients with this disorder."

GPP flares greatly affect a patient’s quality of life and can lead to hospitalization with serious complications, including heart failure, renal failure, sepsis, and death. “The FDA’s action reinforces the potential of spesolimab and represents a critical step in bringing this treatment to patients who need it most,” said Claudia Beqaj of Boehringer Ingelheim, Inc. in a press release. Spesolimab is being investigated further in safety, efficacy, and use in additional GPP settings.

Last year, spesolimab met primary and secondary endpoints in the EFFISAYIL 2 (NCT04399837) trial showing significant prevention of generalized pustular psoriasis (GPP) flares in adolescents and adults for up to 48 weeks.² The positive study results from EFFISAYIL 2 build on data from the previous EFFISAYIL 1 trial (NCT03782792), which demonstrated rapid pustular and skin clearance in patients with GPP flares sustained over 12 weeks. Just last month, permanent J-code (J1747) was released for the spesolimab to manage GPP flares in adults.³ The J-code can be used by commercial insurers and government payers to streamline billing for Medicare Part B drugs.

For more information on GPP and the efficacy of spesolimab, visit our Case-Based Partner Perspectives series featuring Mark Lebwohl, MD, dean for clinical therapeutics at the Icahn School of Medicine at Mount Sinai in New York City and chairman emeritus of the Kimberly and Eric J. Waldman department of dermatology at Mount Sinai.

References

1. Ingelheim B. U.S. FDA Grants Breakthrough Therapy Designation for SPESOLIMAB for the prevention of flares in adults with generalized pustular psoriasis (GPP). PR Newswire: press release distribution, targeting, monitoring and marketing. Published May 2, 2023. Accessed 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

2. A study to test whether BI 655130 (spesolimab) prevents flare-ups in patients with generalized pustular psoriasis. ClinicalTrials.gov identifier: NCT04399837. Updated December 30, 2022. Accessed May 2, 2023. https://clinicaltrials.gov/ct2/show/NCT04399837?term=EFFISAYIL+2&draw=2&rank=1

3. Boehringer Ingelheim Receives Permanent J-Code (J1747) for SPEVIGO® (spesolimab-sbzo) Injection Effective April 1, 2023. Boehringer Ingelheim. Published April 1, 2023. Accessed May 2, 2023. https://www.boehringer-ingelheim.com/us/press-release/boehringer-ingelheim-receives-permanent-j-code-spevigo-spesolimab-sbzo-injection