"Providing Continued Treatment Opportunities
for Existing Clinical Trial Patients After Study Completion"
GemVax & KAEL (GemVax)'s progressive supranuclear palsy (PSP) treatment 'GV1001' has received approval for compassionate use.
According to GemVax on May 21, the Ministry of Food and Drug Safety (MFDS) approved the compassionate use of GV1001 for more than two PSP patients on May 7.
This approval was announced as attention is focusing on the possibility of GV1001's compassionate use (EAP) in the United States, following its recent designation as both an orphan drug and a fast track product by the U.S. Food and Drug Administration (FDA) earlier this month. Compassionate use approval is a system that allows the use of investigational drugs for patients with life-threatening or severe conditions when no alternative treatment options are available.
PSP is a neurodegenerative disease characterized by symptoms such as abnormal eye movement, loss of balance, and decline in speech and cognitive functions. It is similar to Parkinson's disease, but the progression is faster, and there is currently no fundamental treatment, making it considered the most severe among Parkinsonian syndromes.
The approval obtained by GemVax covers use in more than 2 but fewer than 25 patients, allowing physicians to provide GV1001 for compassionate use to PSP patients who wish to continue treatment after participating in the domestic Phase 2 clinical trial and the ongoing extension study. The program will be conducted at Seoul Metropolitan Government Boramae Medical Center, operated by Seoul National University Hospital, and Bundang Seoul National University Hospital.
A GemVax representative stated, "We are pleased that this compassionate use approval will allow PSP patients who participated in clinical trials to continue receiving GV1001 even after the extension study ends," and added, "We will continue to take the lead in expanding treatment opportunities for patients suffering from diseases with no available therapies, both domestically and internationally, through various forms of support."
The representative continued, "We are currently working closely with the FDA and mobilizing all of our resources to expedite the launch of the global Phase 3 clinical trial," and emphasized, "We will do our utmost to succeed in developing the world's first PSP treatment."
Last year, GemVax completed the first-ever domestic Phase 2 clinical trial for PSP. The results showed tolerability and a trend toward slowing disease progression. Based on these results, the company is now pursuing a global Phase 3 clinical trial. In Korea, a 12-month extension study is being conducted for Phase 2 trial patients, and the company plans to confirm the long-term efficacy and safety data for GV1001 over a total of 18 months.
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