Genkyotex’s Systemic Sclerosis Therapy Granted Orphan Drug Designation

Genkyotex, a company focused on developing therapies for hard-to-treat chronic diseases, recently announced that the company’s lead product candidate GKT137831, a drug developed for systemic sclerosis treatment, has been granted Orphan Drug designation by both the U.S. Food and Drug Administration (FDA) and its counterpart in Europe, the European Medicines Agency (EMA).

The Orphan Drug designation is typically given to biologics or drugs whose purpose is to treat rare disorders or conditions. In the United States, a rare condition is defined as a disease that affects less than 200,000 individuals, while in Europe, it is defined as a disease affecting no more than 5 in 10,000 individuals. The Orphan Drug designation provides the sponsor incentives, including protocol assistance during clinical development of the compound, and market exclusivity for 10 years in the U.S. and 7 years in Europe after the drug has been approved.

The therapies developed at Genkyotex are based on selective inhibition of NOX enzymes, which are enzymes known to oxidize proteins, activating several mechanisms and pathways linked to diseases. The inhibition of NOX enzymes prevents the activation of harmful pathways, offering a therapeutic benefit in several medical conditions. GKT137831 is an inhibitor of NOX 1 and 4.

“Systemic sclerosis or scleroderma is one of several fibrotic diseases under evaluation with GKT137831. We believe that NOX inhibition has the potential to dampen multiple biological pathways causing the skin and internal organs of affected patients to undergo fibrosis,” said Philippe Wiesel, MD, Executive Vice President and Chief Medical Officer at Genkyotex, in a press release. “The Orphan Drug designation will allow us to accelerate the clinical assessment of GKT137831 in scleroderma, and to hopefully deliver a much needed therapeutic option for this severe, and life-threatening condition.”

The designation was granted based on results from preclinical studies using several models of fibrotic disorders, including scleroderma. U.S. and EU regulators found GKT137831 to potentially offer a significant benefit to patients with systemic sclerosis, with preclinical data in experimental models showing that its potential to reduce fibrosis (anomalous growth of connective tissue) and improve survival. Furthermore, in a recent Phase 2 clinical trial, treatment with GKT137831 induced a statistically significant reduction in both inflammatory markers and liver enzymes. GKT137831 also showed a solid safety profile in both Phase 1 and Phase 2 clinical trials.