Humanwell Subsidiary Voluntarily Withdraws BLA for Class 1 Gene Therapy Drug pUDK-HGF

Drug Name:Recombinant Plasmid-Hepatocyte Growth Factor Injection

Dosage Form:Injectable

Application Items:Marketing Authorization for Domestically Produced Drugs

Registration Classification:Therapeutic Biological Products Category 1

Applicant:Wuhan Optical Valley Humanwell Biological Pharmaceutical Co.Ltd.

Review Conclusion:Agree to withdraw the registration application of this product and terminate the registration process.

Product Overview:Recombinant Plasmid-Hepatocyte Growth Factor Injection (hereinafter referred to as "pUDK-HGF") is suitable for treating limb rest pain caused by severe lower limb ischemic disease. pUDK-HGF is a novel Class 1 therapeutic biological product.Drug, currently there is no similar product available on the market in China.

R&D Investment:As of now, the cumulative R&D investment in the pUDK-HGF development project is approximately 160 million yuan.

According to the company's description, 20247Month12Day, China National Medical Products Administration Center for Drug Evaluation (CDE) Official website announcement, competitor Northland has submitted its1The new drug application for the innovative drug Sedominje Injection has been submitted and approved.CDEAccepted. Its treatment of lower limb ischemic diseasesⅢPhase Clinical Trial for Resting Pain Indication Unblinded as Planned, Primary Endpoint Met as Expected.

Although the specific technical or review details of this withdrawal were not disclosed in detail according to the company's announcement, it was emphasized that this decision was made after "careful research" by the company.From the perspective of industry practice, it is possible to encounter situations in the review stage where applications are voluntarily withdrawn to supplement or improve data during the new drug application process.. It is widely speculated in the industryIn the case where NL003 has clearly taken the lead, if there are any deficiencies in the materials submitted by Humanwell Healthcare Group Co. Ltd., the difficulty of obtaining direct approval will significantly increase.

Rather than passively waiting for possible unfavorable outcomes, it is better to proactively withdraw and spend the time refining the application materials to prepare for more stringent review comparisons.

The company clearly stated that this withdrawal does not mean the termination of the project.The follow-up plan isResubmit after fully evaluating and improving the research materials according to regulatory and technical requirements.。

Public information shows that the above two drugsBoth are HGF-based naked plasmid gene therapy drugs,Is a treatment for severe lower limb ischemia and other diseasesNew Gene Therapy: The Mechanism of Action Can Be Generally Summarized As:

Main technical features and values include:

• Relatively High Safety: The absence of viral vectors avoids potential risks such as immune reactions and insertional mutagenesis that viruses may cause.

• Long-Lasting and Localized Effects: After entering the cell, the plasmid can persist for an extended period (without integrating into human chromosomes) and continuously express the HGF protein for weeks to months, providing long-term therapeutic effects. The injection takes effect locally with minimal systemic impact.

• Strong Targeting: Directly inject "gene drugs" into the affected area, which is very suitable for localized diseases such as lower limb ischemia.

• Represents the frontier direction: This is an important exploration of gene therapy expanding from addressing rare diseases to tackling common and significant diseases such as coronary heart disease and lower limb ischemia.

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