SPEVIGO® granted Breakthrough Therapy Designation in the U.S. for the prevention of generalized pustular psoriasis flares 

Ingelheim, Germany, 02 May 2023 - Boehringer Ingelheim announced today that spesolimab (marketed as SPEVIGO^®) received Breakthrough Therapy Designation (BTD) as an investigational treatment for the prevention of flares in adolescents and adults with generalized pustular psoriasis (GPP) from the U.S. Food and Drug Administration (FDA).  

“GPP flares may appear suddenly, intensify quickly, and can be life-threatening if left untreated, leaving those affected feeling anxious and uncertain about their future” said Carinne Brouillon, Member of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “The FDA’s recognition of the urgent need for preventing GPP flares is a major step towards empowering people living with the condition to plan critical moments in their lives, despite their disease.” 

This designation follows the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA), who also recently awarded spesolimab a BTD for the prevention of GPP flares. 

The U.S. FDA and China’s NMPA granted these designations based on the topline results from the EFFISAYIL™ 2 trial which studied whether long-term treatment with the antibody spesolimab helps prevent GPP flares in adolescents and adults with GPP up to 48 weeks.^1,2,3 Safety data were in line with previously conducted clinical trials with spesolimab. Data from the trial will be presented at the 25th World Congress of Dermatology 2023 in early July. 

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.^4,6,11 Spesolimab has been approved by regulatory authorities in almost 40 countries including the US, Japan, Mainland China, and the European Union to treat GPP flares in adults.^8,9 

It is the first approved treatment for GPP flares that specifically targets the IL-36 pathway and that has been evaluated in a statistically powered, randomized, placebo-controlled trial.¹¹ Spesolimab is also under investigation for the treatment of other IL-36 mediated skin diseases.¹⁰ 

About the EFFISAYIL^TM clinical trial program 

The EFFISAYIL™ clinical trial program includes: 

• EFFISAYIL™ 1: A Phase II study that demonstrated treatment with a single intravenous dose of spesolimab achieved rapid pustular and skin clearance in patients with GPP flares, sustained over 12 weeks.¹¹ These results supported the approval of spesolimab (SPEVIGO^®) as the first specific treatment for GPP flares in adolescents and adults in major markets. ^8,9,11 

• EFFISAYIL™ 2: A multicenter, randomized, double-blind, placebo-controlled Phase IIb study evaluating the efficacy and safety of maintenance treatment with subcutaneous (SC) spesolimab for the prevention of GPP flares and sustained control of GPP symptoms in adolescents and adults.^1,2,3 

• EFFISAYIL™ ON: An open-label, extension study to evaluate the long-term safety and efficacy of spesolimab in patients with GPP, who have completed previous spesolimab trials.⁵ 

About generalized pustular psoriasis (GPP) 

GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.^4,12 GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body.^4,12 The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.¹² 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.¹³ GPP has a varied prevalence across different geographical regions and more women are affected than men.^4,13-16 There is a high unmet need for treatments with a favorable safety profile that can prevent the occurrence of GPP flares.^11,17 

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.^4,17 

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, sustainable perspective. More than 53,000 employees serve over 130 markets in the two business units Human Pharma and Animal Health. 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. Morita A, Choon SE, Bachelez H et al. Design of Effisayil™ 2: A randomized, double-blind, placebo-controlled study of spesolimab in preventing flares in patients with generalized pustular psoriasis. Dermatol Ther (Heidelb) (2023) 13:347–359. 

2. A study to test whether BI 655130 (spesolimab) prevents flare-ups in patients with generalized pustular psoriasis. www.clinicaltrials.gov/ct2/show/study/NCT04399837?term=Boehringer. Last accessed January 2023. 

3. Spesolimab meets primary and key secondary endpoint for prevention of generalized pustular psoriasis flares. Available at: https://www.boehringer-ingelheim.com/human-health/skin-diseases/gpp/spesolimab-meets-key-endpoint-for-gpp-flare-prevention. Last accessed April 2023.  

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

5. Effisayil ON: An open-label, long term extension study to assess the safety and efficacy of spesolimab treatment in patients with generalized pustular psoriasis (GPP). https://clinicaltrials.gov/ct2/show/NCT03886246?term=Effisayil&draw=2&rank=1. Last accessed January 2023. 

6. Furue K, et al. Highlighting Interleukin-36 Signalling in plaque psoriasis and pustular psoriasis. Acta Derm Venereol. 2018;98:5–13. 

7. Bachelez H, et al. Inhibition of the Interleukin-36 Pathway for the Treatment of Generalized Pustular Psoriasis. N Engl J Med. 2019; 380:981-983. 

8. Federal Drug Administration. New Drug Approvals for 2022. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761244s000lbl.pdf. Accessed January 2023. 

9. EC approval statement (data on file).  

10. A study to test whether spesolimab helps people with a skin disease called hidradenitis suppurativa. Available at: https://clinicaltrials.gov/ct2/show/NCT04762277. January 2023. 

11. Bachelez H et al. Trial of spesolimab for generalized pustular psoriasis. NEJM. 2021;385:2431-40. 

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

13. 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. 

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

15. 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. 

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

17. Puig L, Choon SE, Gottlieb AB, et al. Generalized pustular psoriasis: a global Delphi consensus on clinical course, diagnosis, treatment goals, and disease management. J Eur Acad Dermatol Venereol. 2023 Jan 6. doi: 10.1111/jdv.18851.