New Data Published on Celiprolol for vEDS

In a recent press release, pharmaceutical company Acer Therapeutics, Inc. (“Acer”) announced the publication of data on celiprolol for patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). The article, published in the European Journal of Vascular and Endovascular Surgery, followed 40 patients with vEDS over an 8-year period. Ultimately, this data provides more insight into how celiprolol (EDVISO) changes patient outcomes.

Celiprolol

Also known as EDVISO, celiprolol is a new molecular entity (NME). The FDA defines NMEs as:

[containing] active moieties that have not been approved by FDA previously, either as a single ingredient drug or as part of a combination product; these products frequently provide important new therapies for patients.

Prior to the publication of this data, celiprolol has been granted both Orphan Drug and Priority Review designations. However, the FDA did explain that additional clinical trials are needed to determine how celiprolol reduces the risk of clinical events related to vEDS. Although Acer appealed this decision, the FDA has asked the company to explore additional lines of research prior to resubmitting the company’s New Drug Application (NDA).

Currently, there are no approved treatments for patients with vEDS. Unfortunately, this is considered the most severe subtype of EDS; patients may experience intestinal, uterine, or arterial ruptures, which may be fatal. The average age of death for patients with vEDS is around 51 years old. Thus, finding a potential treatment is necessary to fit this unmet need.

The recently published data is based off of a study of patients from Uppsala University Hospital. Patients were monitored for anywhere from 1 to 98 months, with an average time of 22 months. During this study, the 40 patients received celiprolol 2x daily, with a maximum overall dosage of 400mg. Researchers determined that the drug was fairly well-tolerated. However, vascular issues did arise. Out of the 40 patients, 5 (12.5%) experienced a serious vascular event, 4 of which were fatal.

Ehlers-Danlos Syndrome (EDS)

Overall, Ehlers-Danlos syndrome (EDS) is an umbrella term for a group of inherited disorders which impact connective tissue of the skin, bones, organs, blood vessels, and body tissue. EDS affects collagen and related protein; as a result, bodily tissue experiences changes in structure and functionality. There are an estimated 13 subtypes of EDS, including vascular EDS (vEDS), myopathic EDS (mEDS), and arthrochalasia EDS (aEDS). Although these subtypes vary in genetics and symptoms, they often share similar characteristics of hypermobility, stretchy skin, and fragile tissues. An estimated 1 in every 2500-5000 people has EDS. While symptoms vary, they may include:

  • Pregnancy complications
  • Soft, velvety skin that is hyper-elastic and extremely fragile
  • Easy bruising and bleeding
  • Poor healing after scars
  • Hypermobility
  • Muscle tears
  • Finger, neck, and spine deformities
  • Internal bleeding
  • Stroke
  • Fatigue
  • Heartburn
  • Constipation
  • Frequent joint dislocation
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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