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 Rapid Commercialization Possible if Phase 2 Succeeds

– Overseas Applications, Including to FDA, Planned

GemVax & KAEL Co., Ltd. (GemVax, 082270) announced on the 23rd that its progressive supranuclear palsy (PSP) treatment, 'GV1001,' was designated as a 'development-stage orphan drug' by the Ministry of Food and Drug Safety on the 22nd.

The orphan drug designation system supports the expedited development and approval of treatments for rare diseases. In Korea, it applies to medicines for conditions affecting fewer than 20,000 patients, where no current treatments exist, or where the new drug shows greater safety and efficacy than existing options.

With orphan drug designation, GV1001 is now eligible for the fast-track review process, potentially allowing it to be marketed with ‘conditional approval’ after only Phase 2 clinical trials.

This designation paves the way for GemVax to apply for conditional approval upon confirmation of efficacy in domestic Phase 2 trials. Other benefits include extended product license validity and market exclusivity. GemVax also plans to pursue orphan drug status overseas, including from the FDA.

PSP, a rapidly progressing atypical Parkinson’s syndrome, is the most severe form of Parkinsonism with limited treatment options, making the development of new therapies urgent.

GemVax is currently conducting its first domestic Phase 2 clinical trial for PSP, having successfully enrolled 66 patients (88% of target). Global efforts are also advancing, with the U.S. FDA approving a Phase 2 clinical trial on the 9^th (local time).

GV1001, a multi-mechanism drug, acts on various telomerase functions and is expected to improve the brain's immune environment and reduce inflammation in neurodegenerative diseases like PSP and Alzheimer’s disease. In preclinical PSP trials, GV1001 showed statistically significant improvements in motor and spatial cognitive functions and inhibited tau protein damage.

A GemVax official stated, “This orphan drug designation will be invaluable in our efforts to develop the world’s first PSP treatment. We are fully committed to delivering effective treatments for PSP patients who currently have no options, through successful domestic and global clinical trials.”

The global orphan drug market is expanding rapidly. According to data from the Korea National Enterprise for Clinical Trials (2023), the global orphan drug market is expected to reach approximately $300 billion (₩398 trillion) by 2028. This represents an average annual growth rate of 11.6%, significantly outpacing the 6.1% growth rate of the overall prescription drug market (excluding generics and orphan drugs), which is projected to grow from $922 billion (₩1,225 trillion) to $1.24 trillion (₩1,647 trillion) during the same period.

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