Acer Therapeutics submitted a new drug application (NDA) seeking the approval of its investigative therapy Edsivo (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS) to the U.S. Food and Drug Administration (FDA).
Acer requested priority review and, if granted, the FDA will have 60 days to accept or reject the application. Priority review is given to therapies that promise to significantly improve current treatments.
In the study, participants received either 200 mg of oral Edsivo twice daily (25 patients) or a placebo (28 patients) for up to five years.
The results showed that patients taking the placebo had a higher rate of serious cardiovascular events compared to those taking Edsivo — 50% of the placebo group (14 patients) developed rupture or leak of an artery, versus 20% (five patients) taking Edsivo.
The results suggest that Edsivo reduced the risk of having a serious cardiac event by 64%.
Edsivo-treated patients were also protected from intestinal or uterine rupture, with 24% developing these events compared with 61% in the placebo group.
During the study, patients reported few side effects related to the treatment, with fatigue being the most common.
“Our NDA submission represents the culmination of extensive efforts of our employees, investigators, clinical trial sites, contract research organizations, caregivers and patients,” William Andrews, MD, chief medical officer of Acer, said in a press release.
“We now look forward to continuing to work with the FDA as they review our NDA, with hopes to make Edsivo available as quickly as possible in the U.S. We are grateful to the vEDS patient and advocacy community for their continued involvement, support and feedback as we work together to advance Edsivo, which has the potential to be a significant step forward in the care of patients with this devastating disease,” Andrews said.
vEDS is the most severe subtype of Ehlers-Danlos syndrome, characterized by faulty collagen production due to mutations in the COL3A1 gene.
Edsivo works by promoting the production of collagen in blood vessels, thereby reducing the pressure in major vessels most prone to rupture. The therapy was initially developed to treat high blood pressure, but given its mode of action, it shows promise as a therapy for vEDS.
The therapy received the FDA’s orphan drug status in 2015 for the treatment of vEDS, a designation that helped foster its clinical development.