RARE Daily

FDA Grants Breakthrough Therapy Designation for Spesolimab for the Prevention of Flares in Adults with Generalized Pustular Psoriasis

May 3, 2023

Rare Daily Staff

The U.S. Food and Drug Administration granted Breakthrough Therapy designation for Boehringer Ingelheim’s spesolimab as an investigational treatment for the prevention of flares in adults with generalized pustular psoriasis.

Generalized pustular psoriasis (GPP) is a rare, heterogenous, and potentially life-threatening chronic neutrophilic skin disease characterized by flares of widespread eruptions of painful, sterile pustules across the body. 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. Flares greatly affect a person’s quality of life and can lead to hospitalization with serious complications, including heart failure, renal failure, sepsis, and death. GPP has a varied prevalence across different geographical regions and more women are affected than men.

“GPP flares can appear suddenly and intensify quickly, often leaving patients with a heavy emotional burden,” said Claudia Beqaj, executive director, Dermatology, Sales and Marketing, Boehringer Ingelheim. “The FDA’s action reinforces the potential of spesolimab and represents a critical step in bringing this treatment to patients who need it most. We look forward to working with the FDA to accelerate the development of this treatment.”

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a medicine that is intended to treat a serious or life-threatening disease and preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available treatments. Breakthrough Therapy designation is supported by results from the Effisayil 2 trial which studied spesolimab in the prevention of flares in adults with generalized pustular psoriasis.

Spesolimab is marketed as Spevigo (spesolimab-sbzo) injection, for intravenous use by Boehringer Ingelheim Pharmaceuticals and is indicated for the treatment of GPP flares in adults. Spesolimab is being investigated for use in additional GPP settings. The safety and efficacy of spesolimab in these settings has not been established. Spevigo (spesolimab-sbzo) injection, for intravenous use is not approved for any additional uses in adults or pediatric patients.

The most common side effects of Spevigo include feeling tired or weak, nausea and vomiting, headache, and itching or itchy bumps.

Photo: Claudia Beqaj, executive director, Dermatology, Sales and Marketing, Boehringer Ingelheim

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