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GemVax Receives Orphan Drug Designation  from U.S. FDA for Progressive Supranuclear Palsy Treatment

Regulatory Milestone Advances Global Clinical Development Program and Enhances Prospects for First-in-Class PSP Therapeutic

 

GemVax & KAEL Co., Ltd. (KOSDAQ: 082270, “GemVax”) announced on May 2 that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to GV1001, the company’s investigational therapeutic candidate for the treatment for progressive supranuclear palsy (PSP). The designation provides access to comprehensive FDA support programs, which are anticipated to enhance developmental efficiency and accelerate the advancement toward what may become the first approved therapeutic intervention for PSP globally.

The FDA's Orphan Drug Designation program was established to incentivize the development of treatments for rare diseases affecting fewer than 200,000 individuals in the United States. The designation confers multiple regulatory and commercial advantages, including tax credits of up to 25% on qualifying clinical trial expenditures, waiver of New Drug Application fees, and seven years of market exclusivity following regulatory approval.

The secured ODD status will enable the company to leverage these regulatory incentives from the initiation of this pivotal clinical phase. The designation follows the company's receipt of development-stage orphan drug status from the Republic of Korea's Ministry of Food and Drug Safety in the previous year.

GemVax has established a robust clinical foundation for GV1001 through the successful completion of Korea's inaugural Phase 2 clinical trial in PSP patients. The study demonstrated favorable tolerability profiles while indicating potential efficacy in slowing disease progression. Building upon these encouraging results, the company is advancing preparations for a comprehensive global Phase 3 clinical trial program.

Progressive supranuclear palsy represents a rare and devastating form of atypical parkinsonism characterized by progressive deterioration in balance, ocular motility, speech, and cognitive function. While sharing certain clinical features with Parkinson's disease, PSP demonstrates a significantly more rapid progression rate and poses substantial challenges for patient management. The absence of disease-modifying therapeutic options represents a critical unmet medical need within the neurological community.

GV1001 is a synthetically derived peptide compound based on the amino acid sequence information of human telomerase. Preclinical studies have demonstrated the compound's diverse biological activities, including antioxidant capabilities and cytoprotective effects. Of particular relevance to neurodegeneration, GV1001 has shown potential to modulate the brain's inflammatory microenvironment and attenuate pathological immune responses, positioning it as a promising therapeutic candidate for neurodegenerative conditions including Alzheimer's disease and PSP.

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