GemVax Receives Treatment Purpose Use Approval for GV1001 in PSP Patients (2 or More)

On May 21, GemVax & KAEL announced that its progressive supranuclear palsy (PSP) treatment, GV1001, has received approval for use for treatment purposes from the Ministry of Food and Drug Safety.

Treatment purpose use approval is a system that allows the use of investigational drugs for patients with life-threatening conditions or when no other treatment options are available.

There is growing interest in the possibility of GV1001, which has previously received both orphan drug and fast-track designations from the US Food and Drug Administration (FDA), being made available for expanded access program (EAP) use in the United States.

The approval GemVax received covers between 2 and fewer than 25 patients. Under the judgment of medical professionals, GV1001 can be used for treatment purposes in patients who wish to receive additional doses after participating in the domestic phase 2 clinical trial for PSP and the ongoing extension trial. This will be implemented at Seoul Metropolitan Government Boramae Medical Center, operated by Seoul National University Hospital, and at Seoul National University Bundang Hospital.

A GemVax representative stated, "With this treatment purpose use approval, PSP patients who participated in the clinical trial can continue to receive GV1001 even after the extension trial ends." The representative added, "We will continue to take the lead in expanding treatment opportunities for patients with diseases lacking available therapies, both domestically and internationally, through a variety of support initiatives."

The representative also explained, "We are currently working closely with the FDA and mobilizing all company resources to expedite the initiation of the global phase 3 clinical trial," and added, "We will do our utmost to succeed in developing the world's first PSP treatment."

Last year, GemVax completed the first phase 2 clinical trial for PSP in Korea. The trial results confirmed the tolerability of the drug and indicated a tendency to slow disease progression. The company is now pursuing a global phase 3 clinical trial. In Korea, a 12-month extension trial is being conducted with phase 2 clinical trial patients, and the company will assess the long-term efficacy and safety data of GV1001 over a total of 18 months.

PSP is a neurodegenerative disease characterized by symptoms such as abnormal eye movements, loss of balance, and deterioration of speech and cognitive functions. Although it is similar to Parkinson's disease, PSP progresses more rapidly and, as there is currently no fundamental treatment, it is considered the most severe form among Parkinsonian syndromes.