Helixmith "FDA Clinical Use Approval for NGENSIS Finished Product Produced by New Process and Formulation"

[Asia Economy Reporter Lee Gwan-joo] Helixmith announced on the 8th that the active pharmaceutical ingredient (DS) and finished pharmaceutical product (DP) of its self-developed gene therapy drug Engensis (VM202) have received clinical use approval from the U.S. Food and Drug Administration (FDA).

In 2018, Helixmith acquired a DS manufacturing facility in San Diego, California, USA, and established Genopis. Under the leadership of the headquarters' process development team, the DS production process was improved. Additionally, to resolve issues with the existing DP, a new formulation and lyophilization process were developed, successfully producing the finished product, enabling commercial mass production.

The products manufactured in this way are considered practically different products by U.S. regulatory authorities, requiring highly sophisticated quality equivalence verification tests. Helixmith proved equivalence through various physicochemical and biological experiments conducted domestically and internationally over three years, submitted the data to the FDA, and received approval for clinical use. This product is scheduled to be used soon in the Phase 3 clinical trial of Engensis (VM202) for diabetic peripheral neuropathy (DPN).

Park Young-joo, Head of Clinical Development and U.S. Corporation at Helixmith, said, “This FDA approval can be regarded as a great achievement. The manufacturing and quality control (CMC) section accounts for about 70% of the Biologics License Application (BLA) review documents, which is much larger in volume and variety than clinical data.” He added, “Many U.S. gene therapy companies face setbacks in the CMC section or experience 2 to 3-year delays in the marketing approval process. This approval will act as a very positive factor for the technology transfer of Engensis (VM202).”

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