Boehringer Ingelheim announces European Medicines Agency’s filing acceptance and validation of marketing authorization application for spesolimab in generalized pustular psoriasis
- The marketing authorization application (MAA) package includes results from the pivotal Effisayil-1 global trial
Ingelheim, Germany, October 29, 2021 – Boehringer Ingelheim announced today that the company’s marketing authorization application (MAA) for the treatment of flares in generalized pustular psoriasis (GPP), has been validated and is now under evaluation with the European Medicines Agency (EMA).
“GPP is a rare, life-threatening neutrophilic skin disease characterized by painful, sterile pus-filled blisters, that can suddenly appear over the body,” said Dr. Janine Lamar, Global Spesolimab Lead at Boehringer Ingelheim. “Despite its name, GPP is very different to the more common plaque psoriasis. With no approved treatments in the EU for GPP flares, acceptance of the application for review of spesolimab brings us one step closer to providing a targeted treatment for people with this distressing, unpredictable and painful skin condition.”
GPP is characterized by episodes of widespread eruptions of painful, sterile pustules (blisters of non-infectious pus).1,2,3 The inflammation can also affect other parts of the body and can lead to infections or other organ complications that may be life-threatening.
There is a high unmet need for treatments that can rapidly and completely resolve the symptoms of GPP flares. Flares greatly affect a person’s quality of life4 and can lead to hospitalization with life-threatening complications, such as heart failure, renal failure, sepsis and even death.5
The marketing authorization application was based on the 12 week Effisayil-1 trial; a multi-center, double-blind, randomized, placebo-controlled trial that evaluated efficacy, safety, and tolerability of spesolimab (single dose 900 mg spesolimab i.v., with the option of a 2nd dose if symptoms persisted on Day 8) in patients experiencing a GPP flare.6 The study demonstrated superiority over placebo in pustular clearance after one week of treatment.
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 several autoimmune diseases pathogeneses, including GPP.1,7,8 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, such as palmoplantar pustulosis (PPP) and hidradenitis suppurativa (HS).9,10
About generalized pustular psoriasis
GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.1,2 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 The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.2 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.5 This chronic, systemic disease has a substantial quality of life impact for patients and healthcare burden. GPP has a varied prevalence across geographical regions and more women are affected than men.1,11,12,13
Treatments for GPP
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. Immunomodulatory therapies, including biologics, are used in the treatment of GPP based on clinical experience in patients with plaque psoriasis. However, there is limited evidence on the efficacy and safety of these therapies in the treatment of GPP.
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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 Crowley JJ, et al. A brief guide to pustular psoriasis for primary care providers, Postgraduate Medicine. 2021;133(3):330-344.
2 Navarini AA, et al. European consensus statement on phenotypes of pustular psoriasis. JEADV. 2017;31:1792-1799.
3 Fujita H, et al. Japanese guidelines for the management and treatment of generalized pustular psoriasis: The new pathogenesis and treatment of GPP. J Dermatol. 2018;45(11):1235–1270.
4 Sampogna F, et al. Measuring quality of life of patients with different clinical types of psoriasis using the SF-36. Br J Dermatol. 2006;154(5):844–849.
5 Jeon C, et al. Generalized pustular psoriasis treated with apremilast in a patient with multiple medical comorbidities. JAAD. 2017;Nov;3(6):495–497.
6 Choon et al. Study protocol of the global Effisayil 1 Phase II, multicentre, randomised, double-blind, placebo-controlled trial of spesolimab in patients with generalized pustular psoriasis presenting with an acute flare. BMJ Open 2021;11:e043666
7 Furue K, et al. Highlighting Interleukin-36 Signalling in Plaque Psoriasis and Pustular Psoriasis. Acta Derm Venereol. 2018;98:5–13.
8 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.
9 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 2021.
10 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: October 2021.
11 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.
12 Augey F, et al. Generalized pustular psoriasis (Zumbusch): a French epidemiological survey. European Journal of Dermatology. 2006; 16(6):669-673.
13 Jin H, et al. Clinical features and course of generalized pustular psoriasis in Korea. The Journal of Dermatology. 2015; 42(7):674-678.
