Edsivo Named Breakthrough Therapy by FDA for COL3A1-positive vEDS
The designation is meant to expedite the development and review of medicines that are intended to treat a serious condition, and for which early clinical evidence indicates that the therapy may be a substantial improvement over existing treatments.
“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 Edsivo to rare disease patients, who are often overlooked or underserved,” Adrian Quartel, MD, chief medical officer of Acer Therapeutics, said in a press release.
Acer is planning to launch a pivotal clinical trial testing Edsivo in people with vEDS. Last year, the company submitted the trial protocol, along with an application seeking permission to conduct clinical testing in humans. The FDA cleared Acer to run clinical testing of Edsivo in January.
The company and the FDA are currently working toward an agreement on a special protocol assessment (SPA), which is a process in which drug developers may ask to meet with the FDA to discuss specific details about the design of planned clinical trials or other studies.
Based on preliminary statistical calculations, Acer estimates that the planned clinical trial would need to enroll 150 COL3A1-positive vEDS patients to have enough statistical power to detect a significant effect of treatment. The study is expected to last about 3.5 years once it is fully enrolled.
If Acer and the FDA reach an agreement on the study’s protocol through the SPA, then the company expects the trial — dubbed Decentralized Study of Celiprolol on vEDS-related Event Reduction, or DiSCOVER — will start by the end of June.
“We look forward to continuing our discussions with FDA, through the SPA process, to seek agreement on the protocol design of the proposed pivotal Phase 3 DiSCOVER trial that we plan to initiate by the end of Q2 2022 once agreement is reached,” Quartel said.
vEDS is characterized by deficient production of collagen, a structural protein, which leads to abnormally weak blood vessels. Originally developed to treat high blood pressure, Edsivo may increase collagen production in blood vessels.
In an earlier Phase 4 trial called BBEST (NCT00190411), which enrolled 53 people with vEDS, treatment with Edsivo was found to reduce the risk of artery, intestinal, and uterine ruptures. Based largely on these findings, Acer asked the FDA to approve Edsivo for vEDS in 2018, but the agency rejected the application, requesting more data on effectiveness. A formal appeal of the rejection was also denied.