Acer to Seek FDA Approval for Edsivo to Treat Severe Form of Ehlers-Danlos
The company expects to submit a new drug application (NDA) to the U.S. Food and Drug Administration by the end of the first half of 2018, following pre-NDA meetings later in this second quarter.
The NDA will be supported by clinical results from a Phase 4 trial (NCT00190411) that included 53 patients with vEDS across eight centers in France and one in Belgium. Participants were randomized to receive 200 mg twice daily of oral (25 patients) Edsivo or a placebo (28 patients) for up to five years.
Results showed that five patients (20%) receiving Edsivo experienced an arterial event (arterial rupture or dissection, fatal or not) during the study period compared with 14 patients (50%) in the placebo group, suggesting that Edsivo reduced the risk of having a serious cardiac event by 64%.
Edsivo was also shown to protect patients from intestinal or uterine rupture, which were reported in 24% of treated patients compared with 61% in the placebo group.
During the study, only a few adverse events related to the treatment were reported, with fatigue being the most common side effect.
“We have continued to make progress towards finalizing the critical activities over the next few months required to submit a New Drug Application (NDA) for Edsivo in vascular Ehlers-Danlos syndrome (vEDS) as well as conducting pre-commercial activities,” Chris Schelling, CEO and founder of Acer, said in a press release.
“We are also active in supporting patient advocacy initiatives and are working very closely with the vEDS community to better understand their needs and to learn how we can best partner with them going forward,” he said.
Previously known as EDS type 4, vEDS is the most severe subtype of Ehlers-Danlos syndrome, characterized by faulty production of collagen due to mutations in the COL3A1 gene.
People with vEDS don’t usually show the typical symptoms of the disease, which include skin that easily stretches and unusually wide range of motion in joints. Because of this, the disease is often only detected after severe complications occur, such as when the colon tears or an artery ruptures due to tissue fragility.
Edsivo was initially developed to treat high blood pressure. Now Acer Therapeutics is evaluating its potential to treat vEDS. The company believes that Edsivo can be beneficial for vEDS patients by promoting the production of collagen in blood vessels, while reducing the pressure in major vessels most prone to rupture.
In 2015, the FDA granted Edsivo orphan drug status as a vEDS treatment. This helps expedite the therapy’s clinical development and review, allowing quicker delivery of this potential therapy to patients.