FDA Grants Breakthrough Therapy Designation to Acer for Vascular Ehlers-Danlos Syndrome Candidate
April 4, 2022
The U.S. Food and Drug Administration granted Breakthrough Therapy designation to Acer Therapeutics’ experimental therapy Edsivo for the treatment of patients with COL3A1-positive vascular Ehlers-Danlos syndrome.
Breakthrough Therapy designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy.
Ehlers-Danlos syndrome (EDS) is an inherited disorder caused by mutations in the genes responsible for the structure, production, or processing of collagen, an important component of the connective tissues in the human body, or proteins that interact with collagen. EDS is a spectrum disorder where patients present with various forms, the most serious of which is vascular Ehlers-Danlos syndrome (vEDS), also known as vEDS type IV, which is generally caused by a mutation in the COL3A1 gene resulting in reduced collagen levels.
vEDS causes abnormal fragility in blood vessels, which can give rise to aneurysms, abnormal connections between blood vessels known as arteriovenous fistulas, arterial dissections, and spontaneous vascular ruptures, all of which can be potentially life-threatening. Gastrointestinal and uterine fragility or rupture also commonly occur in vEDS patients. Spontaneous arterial rupture has a peak incidence in the third or fourth decade of life in vEDS patients but may occur earlier and is the most common cause of sudden death in vEDS patients. Arterial rupture or dissection events occur in about 25 percent of patients before the age of 20 but increase to roughly 90 percent of patients by the age of 40. The median survival age of vEDS patients in the U.S. is 51 years, with arterial rupture being the most common cause of sudden death.
“With no currently approved treatments for vEDS anywhere in the world, this designation by FDA marks an important step forward in support of our goal to provide treatment options like Edviso to rare disease patients, who are often overlooked or underserved,” said Adrian Quartel, chief medical officer of Acer.
In December 2021, Acer submitted to FDA a protocol for its proposed prospective pivotal trial, along with an application to begin human clinical trials, which the FDA approved in January 2022. Acer is working with the agency to reach agreement on a special protocol assessment, a process in which sponsors may ask to meet with FDA to reach agreement on the design and size of certain clinical trials, clinical studies, or animal studies. Based on initial statistical power calculations, the company anticipates that the trial would plan to enroll approximately 150 COL3A1-positive vEDS patients, all in the United States. The duration of the clinical trial is currently estimated to be approximately 3.5 years to completion, once fully enrolled.
If agreement on the design of the protocol is reached with FDA through the special protocol assessment, initiation of the Edsivo Decentralized Study of Celiprolol on vEDS-related Event Reduction (DiSCOVER) pivotal clinical trial is expected by the end of the second quarter of 2022, subject to additional capital required to conduct and complete the trial beyond mid-2022.
Author: Rare Daily Staff
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