GemVax Receives Orphan Drug Designation in Europe Following US and Korea

Various Benefits Including 10 Years of Market Exclusivity in Europe

GemVax & KAEL (GemVax) announced on May 29 that its progressive supranuclear palsy (PSP) treatment, GV1001, has been granted Orphan Medicinal Product (OMP) designation by the European Medicines Agency (EMA).

This follows the early May designation of GV1001 as both an orphan drug and a fast track product by the US FDA, marking another milestone with the orphan drug designation in Europe.

With this, GV1001 has now received orphan drug designation from major new drug regulatory agencies in the United States, Europe, and Korea, enabling the company to benefit from each region's respective incentives for drug development.

The EMA offers several benefits for the development of treatments for rare diseases affecting fewer than 50,000 patients in Europe, including scientific advice and development support, reductions or exemptions in fees for marketing authorization applications and scientific consultations, and ten years of market exclusivity after approval.

Previously, GemVax secured benefits in the United States with the FDA's orphan drug designation, including seven years of market exclusivity after approval and a tax credit of up to 25% for clinical trial costs. On May 7, the company also obtained fast track designation from the FDA, which is expected to further accelerate the clinical development and approval process for its PSP treatment.

GemVax plans to accelerate its global Phase 3 clinical trials, leveraging the benefits and support associated with orphan drug and fast track designations.

A GemVax representative stated, "Receiving orphan drug designation in both the United States and Europe, the most influential pharmaceutical markets in the world, validates both the potential and the necessity of developing this treatment. We will do our utmost to quickly advance global clinical trials utilizing these various benefits, so that we can provide a new treatment option for PSP patients."

GemVax previously confirmed the tolerability and a trend toward slowing disease progression in Korea's first Phase 2 clinical trial for PSP, which was completed last year. In addition to the six-month domestic Phase 2 trial, a 12-month extension study is currently progressing smoothly.

PSP is classified as an atypical parkinsonian syndrome, with its main symptom being "supranuclear palsy," or dysfunction in eye movement due to abnormalities in the brain's neural nuclei. Other symptoms include gait disturbances, tremors, and cognitive decline similar to Parkinson's disease, but PSP progresses more rapidly and, to date, there is no fundamental cure for this neurodegenerative disorder.