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Sixera Pharma Receives Rare Pediatric Disease Designation (RPDD) from FDA for SXR1096 for treatment of Netherton syndrome

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Stockholm, July 5, 2022. The Swedish biotech company, Sixera Pharma (”Sixera”) today announced that the US Food and Drug Administration (FDA) has granted rare pediatric disease designation for the kallikrein inhibitor SXR1096 for the treatment of Netherton syndrome.

SXR1096 is a first-in-class, potent and selective small molecule inhibitor of kallikreins 5, 7 and 14 (KLK5, 7 and 14). The compound is formulated in a proprietary skin cream formulation designed for daily use in patients with rare skin conditions. Due to the inborn lack of the protease inhibitor LEKTI, KLK 5, 7 and 14 are overactive in the outermost layer of the skin in patients with Netherton syndrome. These drug targets can best be reached by applying the pharmaceutical directly on the skin. The disease is manifest from birth and can be life threatening in newborns.

“We are very gartified that SXR1096 has received an RPDD from the FDA. We are currently progressing our first clinical trial with SXR1096 in specialist clinical centers in Europe, and plan to file an Investigational New Drug (IND) with the FDA next year, to support initiation of a trial also in the US” said Sixera CEO, Maarten de Château, M.D., Ph.D. “We constantly speak to parents, patients and caregivers who remind us of the urgent need for a therapy for patients with Netherton syndrome, who have a large unmet medical need for a specific treatment throughout life, but especially in young age”.

For more information, please contact:

Maarten de Chateau, CEO Sixera Pharma, info@sixerapharma.com

Visit our homepage: www.sixerapharma.com

About Netherton syndrome:

Netherton syndrome is an inherited, genetic disease based on mutations in a gene coding for the protease inhibitor called LEKTI, that has a role in regulating the activity of proteases in the outermost layer of the skin, determining the pace at which skin is shedded. Individuals with this disease can have a red and scaling skin, that is dysfunctional – leading to loss of fluid over the skin as well as increased risk of developing allergies and serious infections. Itching is a prominent symptom. The hair is brittle, breaking off easily. Symptoms can vary between individuals and over time. The disease is lifelong. Treatment with SXR1096 is expected to restore the damaged skin barrier.

Currently, there is no targeted treatment for Netherton syndrome. Treatment is focused on alleviating the symptoms and preventing allergies and infections. Treatment with skin creams at least twice a day is a must for most patients.

Rare Pediatric Disease Designation:

A rare pediatric disease is defined by the Federal Food, Drug, and Cosmetic Act to include a serious or life-threatening disease, which primarily affects individuals aged from birth to 18 years and affects fewer than 200,000 people in the U.S. To address the challenges that drug companies face when developing treatments for these unique patient populations, Congress reauthorized the Creating Hope Act. Since first enacted by Congress in 2012, the Creating Hope Act has been extended multiple times, most recently on December 27, 2020. Under this Act, companies are eligible to receive a Priority Review Voucher (PRV) following approval of a product with an RPDD if the marketing application receives FDA approval by September 30, 2026, under current legislation, or at a later date if the Act is extended by Congress. If issued, a sponsor may redeem a PRV for priority review of a subsequent marketing application for a different product candidate, or the PRV can be sold or transferred to another sponsor.

About Sixera Pharma: Sixera Pharma is a dermatology company, developing a novel treatment for the orphan indication Netherton syndrome. The company has developed a series of patent protected compounds that are specific inhibitors of the proteases active in the skin; Kallikreins 5, 7 and 14. The company has also developed a proprietary cream formulation of the drug that is easy to apply.

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