FDA puts experimental therapy that blocks HAE swelling on fast track
Designation could accelerate Phase 3 clinical testing of RNA drug onvuzosiran
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Onvuzosiran, an experimental treatment for hereditary angioedema (HAE) now in Phase 3 testing, has been granted fast track designation from the U.S. Food and Drug Administration (FDA).
The FDA grants this status to expedite the development and review of experimental medicines that show potential to address unmet medical needs for serious conditions. For developer Adarx Pharmaceuticals, the status offers advantages such as more frequent meetings with the FDA throughout development and eligibility for priority review if certain criteria are met.
“We are pleased that the FDA has granted Fast Track designation for onvuzosiran, based on the data generated to date and reflecting the significant unmet need and treatment burden faced by people living with HAE,” Zhen Li, PhD, president and CEO of Adarx, said in a company press release. “As we continue to progress the Phase 3 trial, this marks an important milestone for the program.”
HAE is a genetic disease marked by elevated levels of bradykinin, a signaling molecule that can cause fluid to leak into tissues. High bradykinin levels drive the swelling attacks that characterize HAE.
Bradykinin’s production is mediated by an enzyme called kallikrein. Onvuzosiran, also known as ADX-324, is an RNA-based therapy designed to prevent the production of a precursor of the kallikrein enzyme. By limiting the production of the precursor, onvuzosiran aims to lower kallikrein levels and activity, thereby decreasing bradykinin production and ultimately preventing or reducing the frequency of swelling attacks.
Phase 3 trial actively recruiting participants
In a Phase 1/2 clinical trial (NCT05691361), onvuzosiran was shown to reduce kallikrein levels as designed.
Adarx is now sponsoring a Phase 3 clinical trial, STOP-HAE (NCT06960213), to further assess the safety and efficacy of onvuzosiran. The study aims to recruit about 90 adults with HAE type 1 or 2. Participants will be randomly assigned to receive onvuzosiran at one of two doses or a placebo. The main goal is to measure how effectively the therapy reduces monthly swelling attacks over six months.
The STOP-HAE trial is currently recruiting participants at study sites worldwide. To qualify, patients must have had at least one investigator-confirmed swelling attack during the first month of screening, or two attacks within two months, and be able to use on-demand treatment to manage swelling attacks, among other criteria.
The FDA has previously granted onvuzosiran orphan drug designation, which aims to incentivize the development of treatments for rare diseases.
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