Acer Therapeutics has submitted a New Drug Application to the United States Food and Drug Administration on behalf of Edsivo™, their experimental treatment for vascular Ehlers-Danlos syndrome. To find out more, click here to view the source press release at Globe Newswire.
About Vascular Ehlers-Danlos Syndrome
Ehlers-Danlos syndrome is a group of inherited disorders that affect the connective tissue. Vascular Ehlers-Danlos syndrome (vEDS) is a sub-type of this group. It is generally considered to be the most severe form and is often life-shortening. The symptoms of vEDS vary between patients, but many people experience very fragile tissues (including arteries, muscles, and organs) that are at risk of rupture. People with the condition may also have hypermobile joints, thin, translucent skin, and tend to bruise easily. To find out more about vEDS, click here.
Edsivo™ is an investigational drug being developed as a potential therapy for people with vEDS. According to Acer’s website, it is believed to work by promoting normal collagen synthesis in blood vessels, as well as reducing the pressure in vessels prone to rupture. Edsivo™ has been granted Orphan Drug Designation by the FDA.
Research into Edsivo™(celiprolol)
In one study of the drug, 53 people diagnosed with vEDS were divided into two groups, one of which was treated with celiprolol, and one which was a control group. Data showed that 20% of those in the treatment group had an arterial event (rupture or dissection), whereas arterial events occurred in 50% of the control group, supporting the effectiveness of the drug.
The New Drug Application (NDA)
NDAs are the way drug sponsors formally ask the FDA to approve a new drug for sale and marketing within the US. NDAs use the data collected from the research and clinical trials of the drug, which gives the FDA information to base their decision on. The FDA considers a range of factors, such as whether the drug is safe, effective, its benefits and risks, and what the appropriate labelling should be, amongst others. The FDA typically has sixty days to decide whether to accept the NDA submission for review. You can find out more about NDAs here. In addition to the NDA, Acer has also applied for Priority Review, which would shorten the review period.