FDA Accelerated Approval Revision... GemVax Expects Early Commercialization of World's First PSP Treatment

Recently, the Food and Drug Administration (FDA) announced new guidelines regarding accelerated approval, drawing attention from pharmaceutical companies developing new drugs.

GemVax & Kael announced on the 16th that it will establish a development strategy to expedite the development and market entry of GV1001, a treatment for progressive supranuclear palsy (PSP). PSP is a rare disease with no treatments developed worldwide to date.

GemVax plans to actively utilize the accelerated approval pathway to shorten the clinical trial period typically required in global Phase 3 clinical trials or to devise measures for early market entry. The company is focusing on building a strategy for the rapid market entry of PSP treatments in the United States.

On the 6th, the FDA released a draft of new guidelines for the Accelerated Approval pathway. Accelerated approval is a regulatory review system designed to quickly provide new, safe, and effective drugs to patients with life-threatening or serious diseases when there are no meaningful treatment alternatives.

GemVax received news about the new accelerated approval guidelines from Dr. Christophe Diaz, Chief Officer of CurePSP, a nonprofit charitable organization based in the United States.

A notable point in the draft of the new guidelines is the description of the clinical endpoint, one of the evaluation variables considered as criteria for accelerated approval, as an "intermediate clinical endpoint" that predicts the intended clinical benefit of the drug.

The intermediate clinical endpoint refers to a therapeutic effect measurable earlier than irreversible disease progression or death (IMM). It is used when the drug’s effect on IMM or other clinical benefits can be reasonably predicted. This was not actively adopted in previous guidelines.

PSP is a fatal or irreversible severe disease and qualifies for accelerated approval. In the case of GV1001, recent results from a domestic PSP Phase 2a clinical trial demonstrated the potential to delay disease progression and provide fundamental treatment, leading to plans for a global Phase 3 clinical trial, making it expected to utilize the accelerated approval pathway.

GemVax plans to actively leverage the FDA’s fast drug development support programs to strengthen the strategy for the global Phase 3 clinical trial and establish a cooperative system with the FDA.

A GemVax representative said, "As a company considering various methods to provide new treatment options to PSP patients as soon as possible, the new accelerated approval guidelines are welcome news." They added, "We will do our best to succeed in developing the world’s first PSP treatment through various measures to improve clinical trial efficiency and expedite market entry."

In addition to the accelerated approval pathway, the FDA operates expedited programs such as Fast Track, Breakthrough Therapy, and Priority Review. Through these expedited programs, the FDA approved 37 new drugs (74%) in 2021, 24 (65%) in 2022, and 36 (65%) in 2023. Drugs approved via the accelerated approval pathway accounted for 14 (16%) in 2021, 6 (16%) in 2022, and 9 (16%) in 2023. It is expected that more drugs will be approved following the revision of the guidelines.