According to a story from BioPortfolio, the pharmaceutical company Acer Therapeutics, Inc., recently announced the company’s New Drug Application (NDA) has been accepted by the US Food and Drug Administration (FDA) for review. This application is for the company’s drug EDSIVO as a treatment for vascular Ehlers-Danlos syndrome (vEDS). The application has been given Priority Review with an estimated review date of June 25th of this year. Acer Therapeutics is dedicated to the development of therapies for rare diseases with critical unmet needs.
About Ehlers-Danlos Syndrome
Ehlers-Danlos syndrome is a group of disorders that affect connective tissue. There are several different types which have varied symptoms and are linked to different genetic mutations. These mutations may be inherited or may appear early in a patient’s development. Symptoms can include loose joints, stretchy skin, scarring, pain, aoritic dissection, scoliosis, and osteoarthritis. Vascular Ehlers-Danlos syndrome is linked to mutations of the COL3A1 gene and is characterized by fragile, transparent skin, thin hair, short stature, fragile organs prone to rupture, distinct facial features, club foot, and joint hypermobility. With no cure currently available, the treatment of these syndromes is supportive and based on symptoms as they appear. Surgery may be utilized to aid joint problems, but results are varied. There is a great disparity in the severity of symptoms; some patients are able to lead fairly normal lives, whereas others may be severely affected. Vascular Ehlers-Danlos syndrome has a median life expectancy of just 48 years. To learn more about Ehlers-Danlos syndrome, click here.
What is Priority Review?
Priority Review is a special designation granted by the FDA which is intended to speed up the process of review for certain drugs. To qualify, a therapy must be supported by data which suggests that it offers significant advantages over current treatment options. Alternatively it must fulfill a currently unmet medical need.
EDSIVO, or celiprolol, is a beta blocker drug which is commonly used as a treatment for high blood pressure. However, a small scale study suggests that it could also be valuable for treating vascular Ehlers-Danlos syndrome. It was able to reduce the frequency of arterial ruptures and dissections. Researchers believe its effect is linked to its ability to promote more normal synthesis of collagen in these vessels and reduce pressure on the vessels most likely to rupture.