The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to Edsivo™ (celiprolol) for the treatment of vascular Ehlers-Danlos syndrome (EDS) in patients with a confirmed type III collagen (COL3A1) mutation.
Vascular EDS is the most severe subtype of Ehlers-Danlos syndrome. The disorder is caused by mutation in the COL3A1 gene, which encodes the chains of type III procollagen. Patients with vascular EDS may suffer from life-threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures.
Edsivo is a β1-adrenoceptor antagonist with partial β2 agonist activity that is intended to help patients with vascular EDS by promoting normal collagen synthesis in the blood vessels, and by shifting the pressure load away from the vessels most prone to dissection and rupture.
In June 2019, the FDA issued a Complete Response Letter to Acer Therapeutics regarding its New Drug Application (NDA) for Edsivo for the treatment of patients with COL3A1-positive vascular EDS. In the letter, the Agency stated that the application could not be approved without additional data from a well-controlled trial.
“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,” said Adrian Quartel, MD, Chief Medical Officer of Acer. “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.”
Acer Therapeutics’ Edsivo™ (celiprolol) granted FDA Breakthrough Therapy designation for vascular Ehlers-Danlos Syndrome. News release. Acer Therapeutics Inc. April 4, 2022. Accessed April 5, 2022. https://www.globenewswire.com/news-release/2022/04/04/2415756/0/en/Acer-Therapeutics-EDSIVO-celiprolol-Granted-FDA-Breakthrough-Therapy-Designation-for-Vascular-Ehlers-Danlos-Syndrome.html