In the United States, the FDA grants Breakthrough Therapy designation to drugs intended to treat serious and life-threatening illnesses, and/or drugs which show significant improvement over available therapies. After receiving this status, the drug developer also earns benefits. These include eligibility for organizational commitment involving senior managers, Fast Track designation features, and intensive FDA guidance. In a news release from April 2022, pharmaceutical company Acer Therapeutics Inc. (“Acer”) shared that its therapy EDVISO (celiprolol) earned Breakthrough Therapy designation. The treatment is designed for those with COL3A1-positive vascular Ehlers-Danlos syndrome (vEDS). Learn more about Breakthrough Therapy designation.

What is EDVISO?

Currently, no approved therapeutic options exist for those with vEDS. Therefore, EDVISO represents a potential path forward to treating patients and improving outcomes within this realm. EDVISO is considered to be a new chemical entity (NCE). Outside of Breakthrough Therapy designation, this therapy also previously earned Orphan Drug designation.

A description from DrugBank Online explains that celiprolol is:

a selective β1 receptor antagonist, a β2 receptor partial agonist and a weak α2 receptor antagonist. [Prior research] demonstrated [that EDVISO, treating vascular Ehlers-Danlos syndrome,] decreased incidence of arterial rupture or dissection.

Currently, Acer is working with the FDA to develop a clinical trial designed to evaluate the safety, efficacy, and tolerability of EDVISO for vEDS. Since the Investigational New Drug (IND) application has been cleared by the FDA earlier this year, Acer can begin working towards the DiSCOVER clinical study. Altogether, Acer hopes to enroll 150 patients with COL3A1-positive vEDS.

About Ehlers-Danlos Syndrome (EDS)

There are multiple forms of Ehlers-Danlos syndrome, an inherited disorder which affects connective tissue, as well as organs and body tissue. For example, some individuals have hypermobile EDS (hEDS), while others have vascular EDS (vEDS). These conditions occur due to inherited defects in collagen and related proteins, which normally play a role in tissue strength, structure, and function. Potential symptoms associated with EDS include:

• Thin, soft, and translucent skin
• Easy bruising and easy skin tearing
• Loose, unstable, and highly flexible joints
• Easy or frequent dislocated joints or sprains
• Finger, neck, and spine deformities
• Poor wound healing
• Premature aged appearance of the hands and feet
• Clubfoot
• Spontaneous lung collapse
• Internal bleeding
• Heart valve disease
• Sudden vessel rupture
• Pregnancy-related complications

Unfortunately, an estimated 90% of individuals with vEDS experience arterial rupture or dissection before 40 years old.