European Commission approves SPEVIGO® (spesolimab) for generalized pustular psoriasis flares

Ingelheim, Germany, December 13, 2022 – The European Commission (EC) granted a conditional marketing authorization for Boehringer Ingelheim’s spesolimab as first in class treatment for GPP flares in adults.¹ Spesolimab 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.^{2, 4-7}

“The symptoms of GPP are devastating and cause immense pain as well as hospitalization during a flare. Increased GPP awareness that leads to accurate and faster diagnosis is one important step to improving the quality of life for people living with GPP,” said Frida Dunger Johnsson, Executive Director, IFPA. “Now with a specifically developed treatment, people living with GPP can be offered a new lease of life by effectively managing the impact of GPP flares.”

“We are delighted to be able to bring this much needed treatment to patients with GPP, whose options were incredibly limited until this year. This approval marks another significant milestone in the continued development of spesolimab in neutrophilic skin diseases which we are investigating in further clinical trials” commented Carinne Brouillon, Member of the Board of Managing Directors, responsible for Human Pharma, Boehringer Ingelheim. “Today’s news marks the first of what we hope will be a number of new treatment options from our accelerated late-stage portfolio with the potential to transform the lives of people for generations to come.”

The EC’s conditional approval of spesolimab is based on results from the pivotal EFFISAYIL^® 1 Phase II clinical trial.² 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 moderate or severe pustulation of the skin. After one week, 54% of patients treated with a single dose of spesolimab showed no visible pustules compared to placebo (6%).² Adverse events were reported in 66% of patients treated with spesolimab and 56% of those receiving placebo after one week.² Infections were reported by 17% and 6% of patients in the spesolimab and placebo groups respectively (at week one).² Serious adverse events were reported in 6% of patients treated with spesolimab (at week one).²

People living with GPP often do not receive a correct or timely diagnosis and may have their symptoms identified as other forms of psoriasis. A Global Delphi Consensus Project classified GPP as phenotypically, genetically, immunologically and histopathologically distinct from psoriasis vulgaris / plaque psoriasis based on a systematic literature review.³ The growing list of regulatory approvals for spesolimab for the treatment of GPP flares underlines that the Delphi panel’s vision of improved diagnosis and treatment for people with GPP is increasingly achievable.

About SPEVIGO^®

SPEVIGO^® (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.^{2, 4-7}

It is the first 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.^8,9

About the EFFISAYIL^® 1 clinical trial²

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.²

After one week, 54% of patients treated with spesolimab showed no visible pustules, compared to 6% of patients treated with placebo.²
After 12 weeks more than 4 out of 5 patients (84.4%) had no visible pustules and clear/almost clear skin (81.3%).⁹

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).²

About generalized pustular psoriasis (GPP)

GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.^{4, 11} GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body.^{4, 11} The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.¹¹ 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.⁴ This chronic, systemic disease has a substantial quality of life impact for patients and places an increased burden on healthcare systems.¹² GPP has a varied prevalence across different geographical regions and more women are affected than men.^{4, 12-14} There is a high unmet need for treatments with an acceptable safety profile that can rapidly resolve the symptoms of GPP flares and prevent their reoccurrence.³

GPP flares can lead to hospitalization with serious complications, including heart failure, renal failure and sepsis, with the unpredictability and severity of these flares greatly affecting a person’s quality of life.⁴ 

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.    EC approval statement (data on file)
2.    Bachelez H et al. Trial of Spesolimab for Generalized Pustular Psoriasis. NEJM. 2021;385:2431-40.
3.    Puig L, et al. Global consensus on the clinical course, treatment and management of generalized pustular psoriasis (GPP). Presented at the European Academy of Dermatology and Venereology (EADV) Congress, Milan, Italy, 7–10 September 2022: P1203.B.
4.    Crowley JJ, et al. A brief guide to pustular psoriasis for primary care providers, Postgrad Med. 2021;133(3):330-344.
5.    Federal Drug Administration. New Drug Approvals for 2022. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761244s000lbl.pdf. Accessed September 2022.
6.    Furue K, et al. Highlighting Interleukin-36 Signalling in Plaque Psoriasis and Pustular Psoriasis. Acta Derm Venereol. 2018;98:5–13.
7.    CHMP positive opinion. Available at: https://www.ema.europa.eu/en/medicines/human/summaries-opinion/spevigo. Accessed November 2022.
8.    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 October 2022. 
9.    Elewski B, et al. Sustained treatment effect of spesolimab over 12 weeks for generalized pustular psoriasis flares; results from the Effisayil 1 study. AAD Congress 2022, Poster 32924.
10.    Navarini AA, et al. European consensus statement on phenotypes of pustular psoriasis. JEADV. 2017;31:1792-1799.
11.    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.
12.    Augey F, et al. Generalized pustular psoriasis (Zumbusch): a French epidemiological survey. Eur J Derm. 2006; 16(6):669-673.
13.    Jin H, et al. Clinical features and course of generalized pustular psoriasis in Korea. J Dermatol. 2015; 42(7):674-678.