Originally posted as a press release.
ResVita Bio, a cellular therapies startup, announces that the FDA has granted Rare Pediatric Disease Designation to RVB-001 to treat Netherton Syndrome, a chronic and life-threatening genetic skin disease.
“The physical and emotional challenges of chronic skin diseases are devastating to patients, particularly children” said Amin Zargar, Chief Executive Officer 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.”
With the Rare Pediatric Disease Designation, if a new drug application for RVB-001 is approved, ResVita Bio may be eligible to receive a priority review voucher, which can be redeemed for priority review of any subsequent drug marketing application. The voucher can also be sold or transferred.
About Netherton Syndrome: Netherton Syndrome is a 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 NS 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.
About RVB-001: 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.
About ResVita Bio: ResVita Bio seeks to restore and maintain the vitality of the skin through topical cellular therapies. Through synthetic biology and metabolic engineering, we are developing a safe and versatile skin probiotic platform to continuously delivers therapies that directly treat inflammatory skin diseases including Netherton Syndrome, atopic dermatitis, chronic wound infection, and psoriasis.