Zevra, nonprofit team up to urge timely testing for life-threatening rare diseases

Zevra Therapeutics has partnered with the National Urea Cycle Disorders Foundation (NUCDF) to run an awareness campaign designed to improve the diagnosis of conditions treated by one of its products.

The Florida-based biotech sells Olpruva for the treatment of urea cycle disorders (UCDs). Awareness of the medicine was low when Zevra acquired it in the $91 million takeover of Acer Therapeutics in August 2023, leading the biotech to seek to raise the product’s profile among physicians who treat the 1,100 patients with UCD in the U.S. Researchers have said UCDs are “largely underdiagnosed worldwide.”

Zevra’s collaboration with the NUCDF could lead to more people being diagnosed. The partners have set up a campaign to raise awareness of the importance of promptly checking blood ammonia levels and of using proper testing techniques.

On the campaign website, checkammonia.com, Zevra and the NUCDF tell healthcare professionals they should perform a plasma ammonia test when a patient has “an unexplained change in consciousness, encephalopathy and with other neurological symptoms.” The website outlines a four-step testing process and advises healthcare professionals to call a metabolic geneticist immediately.

Zevra and the NUCDF have created a frequently asked questions document and a brochure. The texts say the clinical presentation of neonatal hyperammonemia can mimic sepsis. However, when severe symptoms start after 24 hours of age, the cause is typically a UCD or another inborn error of metabolism, according to the campaign, and doctors shouldn’t wait for sepsis results before checking ammonia levels.

The biotech could benefit if the campaign increases the diagnosis of UCDs and raises its profile among physicians who treat the conditions. On an earnings call in August, Zevra CEO Neil McFarlane said the company had “made progress with healthcare providers to increase awareness levels” and “successfully engaged the majority of clinicians and thought leaders who diagnose and treat UCD patients.”

However, McFarlane identified a need to get the word out to patients and to turn awareness into sales. Zevra reported nine new patient enrollments on the UCD drug Olpruva in the second quarter. McFarlane said the number “is not yet where we would like it to be.”