FDA grants orphan drug, rare pediatric disease status to GEn-1123 for DMD
Designations provide incentives for development of treatments for rare disorders
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The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to GEn1E Lifesciences‘ GEn-1123 as a potential treatment for Duchenne muscular dystrophy (DMD).
The designations aim to incentivize the development of treatments for rare disorders, which are those affecting fewer than 200,000 people in the U.S. Orphan drug status provides benefits such as tax credits, fee waivers, and seven years of market exclusivity if the treatment is approved. With the rare pediatric disease status, intended for treatments of serious or life-threatening diseases that primarily affect children, GEn1E may be eligible to receive a priority review voucher that would shorten the FDA’s review time for a subsequent drug application. That voucher can also be transferred or sold to another company.
“Receiving both Orphan Drug and Rare Pediatric Disease Designations for GEn-1123 marks important milestones for GEn1E and further supports our precision medicine approach to address rare and inflammatory diseases,” Ritu Lal, PhD, GEn1E’s founder and CEO, said in a company press release.
GEn-1123 designed to rebalance altered inflammatory signaling
DMD is caused by mutations in the gene that provides instructions for making the protein dystrophin, which normally helps protect muscle cells from wear-and-tear damage during muscle movements. These mutations result in virtually no dystrophin production, leading to muscle weakness and loss of muscle tissue.
The disease is marked by chronic inflammation, impaired muscle regeneration, and muscle scarring (fibrosis), which contribute to the progressive impairment of skeletal, heart, and respiratory function.
Taken orally, GEn-1123 is a novel dual signal modulator designed to rebalance altered inflammatory signaling. According to GEn1E Lifesciences, it does so by reducing pro-inflammatory pathways and promoting anti-inflammatory, pro-survival, and regenerative signaling. It works by selectively blocking the interaction between the p38alpha and MK2 enzymes, which is implicated in inflammation.
We believe GEn-1123 has the potential to become an oral small-molecule differentiated therapy that addresses key drivers of disease progression in patients who remain underserved by current treatment options.
The treatment was developed using the company’s GRID Platform, an artificial intelligence-driven tool that integrates clinical, biomarker, biological mechanisms, and inflammatory pathways information to develop targeted therapies.
“DMD remains a devastating disease for young patients and their families,” Lal said. “We believe GEn-1123 has the potential to become an oral small-molecule differentiated therapy that addresses key drivers of disease progression in patients who remain underserved by current treatment options.”
Another molecule developed by the company to target the same pathway is currently under Phase 2 testing as a treatment for acute respiratory distress syndrome.
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