FDA Grants Rare Pediatric Disease Designation to ResVita’s Potential Treatment for Netherton Syndrome

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to ResVita Bio’s RVB-001 for the treatment of Netherton syndrome, a chronic and life-threatening genetic skin disease.

Netherton Syndrome is a rare skin disorder characterized by chronic skin inflammation, pruritis, and atopic manifestations. Infections and dehydration are common and result in high post-natal mortality, and these disease manifestations often persist throughout a patient’s life. The causative mutations of Netherton syndrome are within the SPINK5 gene, which encodes the serine protease inhibitor LEKTI. These mutations can result in uncontrolled protease activity, leading to premature exfoliation of the outermost layers of the skin.

ResVita Bio uses synthetic biology and metabolic engineering to develop topical cellular therapies to restore and maintain the vitality of the skin. RVB-001 is a genetically engineered probiotic that is topically applied to temporarily colonize the skin and continuously release LEKTI, thereby inhibiting proteolysis and restoring the integrity of the epidermal barrier. 

“The physical and emotional challenges of chronic skin diseases are devastating to patients, particularly children,” said Amin Zargar, CEO of ResVita Bio. “Newborns with Netherton syndrome face a severe lifelong disease with few treatment options that offer limited efficacy. We are motivated by the prospect to deliver meaningful improvement in the lives of these patients, and this is a significant step towards the development RVB-001 as a treatment for Netherton Syndrome.”

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 RVB-001 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA.

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, BridgeBio Pharma sold its priority review voucher for $110 million.

Photo: Amin Zargar, CEO of ResVita Bio