Azitra Receives Rare Pediatric Disease Designation for Netherton Syndrome Treatment

The U.S. Food and Drug Administration granted Azitra Rare Pediatric Disease designation for ATR-12 for the treatment of Netherton syndrome, a chronic genetic skin disease that could be life threatening.

Photo: Richard Andrews, president and CEO of Azitra

Netherton Syndrome (NS) is a rare autosomal recessive disease of the skin, characterized by severe inflammation, pruritus, scaling, redness, and dehydrated skin. It is estimated to affect one of every 200,000 live births worldwide. It has been reported that one in ten infants with NS die in their first year of life. Patients can suffer lifelong challenges including red, scaly skin, hair defects, and an ongoing higher than normal risk for infection and allergy. The disease is caused by mutations in the SPINK5 gene, which encodes the serine protease inhibitor Lympho-epithelial Kazal-type related inhibitor (LEKTI). There are currently few treatment options for NS.

ATR-12 is a proprietary strain of Staphylococcus epidermidis engineered to express therapeutic levels of LEKTI protein to treat NS. The microbiome-based product is designed to reside on the skin and provide continuous delivery of the missing protein. The function of LEKTI is to regulate protease activity in the epidermis. Proteases in the skin play an important role in the shedding of skin cells in a process known as desquamation. When LEKTI is absent or has reduced activity, excess shedding occurs, and the skin is sensitive, open, and appears red and scaly. ATR-12 will be provided as a non-aqueous ointment for topical application.

 “Currently available treatments for NS have limited efficacy and can cause serious side effects. We are hopeful that ATR-12 will offer a new treatment option to patients suffering from this life-threatening disorder,” said Richard Andrews, president and CEO of Azitra. “Obtaining Rare Pediatric Disease Designation is a major milestone for ATR-12, a product candidate based on our core microbiome technology.”

Azitra plans to begin clinical testing of ATR-12 in 2021.

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes ATR-12 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Sarepta sold its priority review voucher to Vifor Pharma $111 million in February 2020.

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.

Author: Rare Daily Staff