Epygenix Receives ODD and RPDD Designations from FDA > Pipeline News

Epygenix Receives ODD and RPDD Designations from FDA

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Based on its interest in treatments for refractory forms of pediatric genetic epilepsy, Young Poong Pharmaceutical has been steadily conducting research in partnership with Epygenix, a biopharmaceutical company based in New Jersey, USA, that focuses on gene-based therapeutics. As a result, the recent candidate substances EPX-100 and EPX-200 have received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA.

'EPX-100' is a drug under development for the treatment of the rare pediatric diseases Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). It has demonstrated efficacy in a zebrafish model that replicates the gene mutation causing DS, suggesting potential to improve daily functioning in patients with DS and LGS. Currently, important clinical trials for the treatment of Dravet syndrome in adults and children are underway, and a Phase 3 trial for Lennox-Gastaut syndrome is scheduled to start in the second half of 2024, with final data expected in the first half of 2026.

On April 30, 2024, Harmony Biosciences, a NASDAQ-listed company, acquired Epygenix for a total of $680 million and plans to rapidly continue clinical trials. The DS treatment market in the United States is projected to be $575.14 million from 2022 to 2027, while the LGS treatment market is expected to be $699.80 million from 2023 to 2028.

Young Poong Pharmaceutical is deeply discussing securing domestic sales priority negotiation rights for approximately 550 domestic patients suffering from a lack of alternative medications despite high seizure frequency, concurrently with the sale of Epygenix.

●Dravet Syndrome (DS)

Characterized by a high frequency and severity of seizures, intellectual disability, and a risk of sudden death, starting from the first year of life. About 85% of DS cases are caused by new loss-of-function (LOF) mutations in the voltage-gated sodium channel gene SCN1A1, with an estimated incidence of 1 in 15,700.

●Lennox-Gastaut Syndrome (LGS)

A drug-resistant epileptic encephalopathy that typically begins between the ages of 3 and 5. The fundamental cause of LGS is unknown, and it is believed to result from a wide range of factors including genetic differences and structural abnormalities of the brain. Patients exhibit multiple seizure types and developmental, cognitive, and behavioral issues. The number of LGS patients in the United States is estimated to be around 48,000.