ResVita Bio Announces RVB-003 granted Orphan Drug Designation for Netherton Syndrome by the FDA

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Apr 03, 2025, 08:47 ET

BERKELEY, Calif., April 3, 2025 /PRNewswire/ -- ResVita Bio, a therapeutics company focused on treatments for skin diseases, today announced that the FDA awarded the Orphan Drug Designation to RVB-003 for the treatment of Netherton Syndrome, a chronic and life-threatening skin disorder. This milestone, following the FDAs previous granting of the Rare Pediatric Disease Designation, underscores the impact of ResVita Bio's new platform for continuous protein therapy—a groundbreaking therapeutic approach designed to deliver sustained drug levels directly to the skin, supporting both greater efficacy and improved safety over other topicals.

Representative images of Netherton Syndrome mice treated daily with either standard of care (left) or RVB-003 (right). Mice treated with RVB-003 show resolution of excessive protease activity, normalization of inflammation, marked reduction in epidermal hyperplasia, and ultimately a restoration of a healthy skin barrier.

Netherton Syndrome is a rare genetic disorder associated with significant morbidity and risk of early mortality. Caused by mutations in the SPINK5 gene, it leads to breakdown of the skin's protective barrier, leaving infants vulnerable to dehydration, infections, and chronic inflammation. Alongside these risks, individuals also experience intense itching and allergic symptoms, making day-to-day life especially difficult from an early age.

RVB-003 is based on ResVita Bio's continuous protein therapy platform, which utilizes genetically engineered bacteria applied topically. This novel approach aims to address the short lifespan of therapeutic proteins by continuously producing them directly on the skin surface, thereby enhancing efficacy and safety profiles. In its ODD submission, ResVita Bio presented the in vitro, ex vivo, and in vivo studies demonstrating RVB-003's treatment of Netherton Syndrome (Figure 1).

With ODD, ResVita Bio is eligible for various incentives from the FDA, including tax credits for clinical research, potential waiver of certain administrative fees, and, upon approval, up to seven years of market exclusivity in the United States. Most importantly, ODD will foster greater collaboration with the FDA, which will help streamline the development of RVB-003 to deliver therapies to this underserved patient population.

"This is an exciting development for our treatment of Netherton Syndrome," said Dr. Amin Zargar, CEO of ResVita Bio. "Coming off our successful FDA INTERACT meeting, we're eager to discuss our finalized Drug Product with the FDA in a Pre-IND meeting during the summer. This Orphan Designation will enable increased communication throughout development, and we are excited to for clinical trials next year."

About ResVita Bio
ResVita Bio is a therapeutics company based in Berkeley, California, dedicated to revolutionizing the treatment of skin diseases through its continuous protein therapy platform. By utilizing genetically engineered bacteria to deliver continuous therapeutic proteins topically, ResVita Bio aims to enhance the efficacy and safety profiles of its therapies, offering new hope for patients worldwide. This development is supported by private investment and the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) awards 1R43AR082240-01 and 2R44AR082240-02.

For more information, please visit www.resvitabio.com

Media Contact:
Amin Zargar
510-458-2297
[email protected]

SOURCE ResVita Bio