CHMP adopts positive opinion recommending approval of new and expanded indications for SPEVIGO®

• The CHMP positive opinion is based on the EFFISAYIL® 2 trial, which showed that no flares were observed after week 4 of SPEVIGO® administered subcutaneously. During the 48-week trial, an 84% reduction of the risk of GPP flares was observed.
• GPP is a rare, chronic, heterogenous neutrophilic inflammatory disease associated with skin flares and systemic symptoms, such as fever, pain, and fatigue.
• SPEVIGO® is currently approved in 51 countries for treatment of GPP flares, and the CHMP opinion follows similar approvals for expanded and new indications in the US and China.

Boehringer Ingelheim today announced that the European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of a new indication of SPEVIGO® (spesolimab) injection for the prevention of generalized pustular psoriasis (GPP) flares in adults and adolescents from 12 years of age; and extension of the approved indication for SPEVIGO® (spesolimab) infusion for the treatment of generalized pustular psoriasis (GPP) flares in adults and adolescents from 12 years of age as monotherapy. SPEVIGO® is a novel, humanized selective IgG1 antibody that binds to interleukin-36 receptor (IL-36R), a key part of a signaling pathway within the immune system shown to be involved in the cause of GPP.

The regulatory authorities’ decisions are based on the positive results of the EFFISAYIL® 2 trial, a 48-week clinical trial that showed that SPEVIGO® significantly reduced the risk of GPP flares by 84%, compared with placebo. In the trial with 123 patients, no flares were observed after week 4 of SPEVIGO® subcutaneous treatment in the high-dose group (n=30). The EFFISAYIL® 2 trial showed a similar incidence of patients with adverse events across spesolimab and placebo treatment arms.

“GPP presents a significant diagnostic challenge for healthcare professionals as it is a highly variable rare disease that is experienced differently by everyone who has it, and it has suffered from a historic lack of treatment options,” said Dr Peter van der Kerkhof, Professor and previous Chairman of the Department of Dermatology, Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands. “Spesolimab’s recent approvals, combined with the CHMP recommendation provides us with the potential for continuous treatment, addressing a significant unmet need.”

Distinct from plaque psoriasis, GPP is a rare, chronic, heterogenous, inflammatory neutrophilic disease associated with painful skin manifestations and systemic symptoms, such as fever, pain, and fatigue. GPP varies widely between individuals living with the condition, with symptoms presenting both above and below the skin. Uncontrolled GPP may require emergency care and can lead to life-threatening complications, such as multi-organ failure and sepsis. GPP’s unpredictable nature can potentially have significant long-term impacts on quality of life for people living with it and leads to fear and anxiety over the disease course.

“The CHMP’s positive opinion on SPEVIGO®, alongside approvals in China and the US, signals a strong shift in treatment, offering people living with the disease the potential for significant improvement of their condition and a better quality of life,” said Carinne Brouillon, Member of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “The potential to prevent GPP flare-ups means a possibility to gain back control over how people affected by the disease live their lives.”

"GPP goes way beyond the skin, it's a relentless and unpredictable disease that can impact every aspect of a person's life,” said Frida Dunger, Executive Director, IFPA (International Federation of Psoriasis Associations). “I want a world where every person with GPP is diagnosed quickly and receives the treatment they need, and this is their right. There is much more to do, but I believe with all stakeholders working together we are headed in the right direction.”

About SPEVIGO®
SPEVIGO® is a novel, humanized, selective antibody that specifically blocks the activation of the IL-36R, a signaling pathway within the immune system shown to be involved in the pathogenesis of several autoinflammatory diseases, including GPP. It is the first targeted therapy for the treatment of GPP and has been evaluated in the largest clinical program specifically for the treatment of patients with GPP.

About generalized pustular psoriasis (GPP)
GPP is a chronic, heterogenous, neutrophilic inflammatory disease associated with skin and systemic symptoms that is distinct from plaque psoriasis. GPP is recognized as a separate clinical entity from other forms of psoriasis, with the IL-36 pathway being a key driver of GPP and triggering response to treatment. Prevalence of GPP varies considerably across geographical regions, ranging from 1.76 to 124 patients per million persons. GPP can become life-threatening (mortality rates ranging from 2% to 16%) due to severe complications, such as multisystem organ failure and sepsis requiring urgent hospital care; many GPP patients also suffer from various comorbidities, which contribute to the ongoing burden for the patient and healthcare systems. GPP symptoms appear unpredictable and present on a continuum, which greatly impacts a patient’s quality of life, and may cause fear and anxiety over the disease course, as well as long-term impacts on quality of life related to work/school, emotional health, social activities, and finances.

About the EFFISAYIL® clinical trial program
The EFFISAYIL® clinical trial program evaluated the largest and broadest population of GPP patients in trials of a therapy specifically targeting the IL-36 pathway for GPP.

• EFFISAYIL® 1: A Phase II study that demonstrated treatment with a single intravenous dose of spesolimab significantly improved signs and symptoms of GPP in patients experiencing a flare, including rapid pustular and skin clearance. These results supported the approval of spesolimab as the first specific treatment for GPP flares in adults in major markets.
• EFFISAYIL® 2: A Phase IIb study that showed subcutaneous spesolimab significantly reduced the risk of GPP flares by 84% over 48 weeks compared to placebo. In the trial with 123 patients, no flares were observed after week 4 of spesolimab SC treatment in the high-dose group (n=30).

• 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.

• EFFISAYIL® REP (ongoing): An open-label, single-arm, Phase IIIb/IV trial to evaluate the efficacy, safety, and the impact of immunogenicity in the treatment of patients with GPP presenting with a recurrent flare following their initial GPP flare treatment with spesolimab IV.

About Boehringer Ingelheim
Boehringer Ingelheim is a biopharmaceutical company active in both human and animal health. As one of the industry’s top investors in research and development, the company focuses on developing innovative therapies that can improve and extend lives in areas of high unmet medical need. Independent since its foundation in 1885, Boehringer takes a long-term perspective, embedding sustainability along the entire value chain. More than 53,500 employees serve over 130 markets to build a healthier, more sustainable, and equitable tomorrow. Learn more at https://www.boehringer-ingelheim.com/.