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Boehringer Ingelheim Announces EMA Filing Acceptance and Validation of MAA for Spesolimab

The marketing authorization application (MAA) package includes results from the pivotal Effisayil-1 global trial.

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).The inflammation can also affect other parts of the body and can lead to infections or other organ complications that may be life-threatening.  

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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.

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.  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).

About generalized pustular psoriasis
GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis. GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body. The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares. 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.

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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Living with fibrotic and inflammatory diseases greatly impacts patients’ lives emotionally and physically. These patients are our guide, partner and inspiration as we redefine treatment paradigms. As a family-owned company, we can plan long-term. Our goal is to discover and develop first-of-their-kind therapies. With a deep understanding of molecular pathways, we are pioneering scientific breakthroughs that target, repair and prevent many fibrotic and inflammatory diseases. By building on long-term external collaborations we strive to bring treatment breakthroughs to patients in the shortest time. We won’t rest until we can give people the chance to live the life they want

Source: Boehringer Ingelheim
Date: Oct 29, 2021
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