U.S. FDA approves first treatment option for generalized pustular psoriasis flares in adults

Ingelheim, Germany, September 2, 2022 –The U.S. Food and Drug Administration is the first regulatory authority to approve spesolimab as a treatment option for generalized pustular psoriasis (GPP) flares in adults, Boehringer Ingelheim announced today. Spesolimab, marketed in the U.S. as SPEVIGO^®, is a novel, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in the pathogenesis of GPP. 

“GPP flares can greatly impact a patient’s life and lead to serious, life-threatening complications,” said Mark Lebwohl, M.D., lead investigator and publication author, and Dean for Clinical Therapeutics, Icahn School of Medicine at Mount Sinai, Kimberly and Eric J. Waldman Department of Dermatology, New York. “The approval of spesolimab is a turning point for dermatologists and clinicians. We now have an FDA-approved treatment that may help make a difference for our patients who, until now, have not had any approved options to help manage GPP flares.”

“This important approval reflects our successful efforts to accelerate our research with the aim to bring innovative treatments faster to the people most in need,” said Carinne Brouillon, Member of the Board of Managing Directors, responsible for Human Pharma, Boehringer Ingelheim. “We recognize how devastating this rare skin disease can be for patients, their families and caregivers. GPP can be life-threatening and until today there have been no specific approved therapies for treating the devastating GPP flares. It makes me proud that with the approval of SPEVIGO^® we can now offer the first U.S. approved treatment option for those in need.”

The FDA’s approval of spesolimab is based on results from the pivotal EFFISAYIL^® 1 Phase II clinical trial.¹ In the 12-week trial, patients experiencing a GPP flare were treated with spesolimab or placebo. Most patients at the outset of the trial had a high, or very high, density of pustules, and impaired quality of life. After one week, 54% of patients treated with spesolimab showed no visible pustules compared to placebo (6%).¹

In addition to the U.S. approval, spesolimab is currently under review by several other regulatory authorities. To date, spesolimab has received Breakthrough Therapy Designation in the U.S., China mainland and Taiwan, Priority Review in the U.S. and China mainland, Orphan Drug Designation in the U.S., Korea, Switzerland and Australia, Rare Disease Designation and fast track in Taiwan, for the treatment of GPP flares. The European Medicines Agency validated the marketing authorization application for spesolimab in GPP in October 2021 and the submission is currently under evaluation.

Distinct from plaque psoriasis, GPP is a rare and potentially life-threatening neutrophilic skin disease, characterized by episodes of widespread eruptions of painful, sterile pustules. Given that it is so rare, recognizing the symptoms can be challenging and consequently lead to delays in diagnosis. 

About spesolimab

Spesolimab is a novel, humanized, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in the pathogenesis of several autoinflammatory diseases, including GPP. ^1-3
It is the first investigational treatment to specifically target the IL-36 pathway for the treatment of GPP flares that has been evaluated in a statistically powered, randomized, placebo-controlled trial. Spesolimab is also under investigation for the prevention of GPP flares and for the treatment of other neutrophilic skin diseases.^4,5

About the EFFISAYIL^® 1 clinical trial¹

EFFISAYIL^® 1 (NCT03782792) was a 12-week Phase II trial investigating patients with a GPP flare (N=53), randomly assigned 2:1 to a single 900 mg intravenous dose of spesolimab or placebo.¹

After one week, 54% of patients treated with spesolimab showed no visible pustules, compared to 6% of patients treated with placebo.¹

Adverse events were reported in 66% of patients treated with spesolimab and 56% of those receiving placebo after one week. Infections were reported by 17% and 6% of patients in the spesolimab and placebo groups respectively (at week one). Serious adverse events were reported in 6% of patients treated with spesolimab (at week one).¹ 

About generalized pustular psoriasis (GPP)

GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.^{2, 6} GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body.^{2, 6} The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.⁶ While the severity of GPP flares can vary, if left untreated they can be life-threatening due to complications such as sepsis and multisystem organ failure.² This chronic, systemic disease has a substantial quality of life impact for patients and increased healthcare burden.⁷ GPP has a varied prevalence across different geographical regions and more women are affected than men.^{2, 8-10} There is a high unmet need for treatments that can rapidly resolve the symptoms of GPP flares and prevent their reoccurrence, with an acceptable safety profile. 

GPP flares can lead to hospitalization with serious complications, including heart failure, renal failure and sepsis, and the unpredictability and severity of these flares greatly affect a person’s quality of life. 

About the Boehringer Ingelheim Human Pharma R&D pipeline

Boehringer Ingelheim has experienced strong momentum through greater investment in Research & Development (R&D), accelerated development of its pipeline and business development opportunities. The company already closed 11 R&D partnership and license deals in the first half of 2022, with a focus on new modalities such as protein degraders, antimicrobial resistance, regenerative medicine, oncology and data science. 

Boehringer Ingelheim has earmarked 25 billion EUR for investments in R&D and an additional 7 billion EUR for new production technologies over the next five years. The company anticipates running eight active human medicines registrational trials by the end of the year, with the potential of bringing 15 new products to patients by 2027.

About Boehringer Ingelheim 

Boehringer Ingelheim is working on breakthrough therapies that transform lives, today and for generations to come. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. More than 52,000 employees serve over 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com.

Intended audiences

This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.

References

1. Bachelez H et al. Trial of Spesolimab for Generalized Pustular Psoriasis. NEJM. 2021;385:2431-40.

2. Crowley JJ, et al. A brief guide to pustular psoriasis for primary care providers, Postgrad Med. 2021;133(3):330-344.

3. Furue K, et al. Highlighting Interleukin-36 Signalling in Plaque Psoriasis and Pustular Psoriasis. Acta Derm Venereol. 2018;98:5–13.

4. ClinicalTrials.gov. A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Hidradenitis Suppurativa. Available at: https://clinicaltrials.gov/ct2/show/NCT04762277. Accessed August 2022. 

5. ClinicalTrials.gov. A Study to Test Long-term Treatment With Spesolimab in People With Palmoplantar Pustulosis (PPP) Who Took Part in Previous Studies With Spesolimab. Available at https://clinicaltrials.gov/ct2/show/NCT04493424. Accessed: August 2022. 

6. Navarini AA, et al. European consensus statement on phenotypes of pustular psoriasis. JEADV. 2017;31:1792-1799.

7. Hanna M, et al. Economic burden of generalized pustular psoriasis and palmoplantar pustulosis in the United States. Curr Med Res Opin. 2021. 37(5):735-742.

8. Ohkawara A et al. Generalized pustular psoriasis in Japan: two distinct groups formed by differences in symptoms and genetic background. Acta Derm Venereol. 1996 Jan;76(1):68–71.

9. Augey F, et al. Generalized pustular psoriasis (Zumbusch): a French epidemiological survey. Eur J Derm. 2006; 16(6):669-673.

10. Jin H, et al. Clinical features and course of generalized pustular psoriasis in Korea. J Dermatol. 2015; 42(7):674-678.