Retrophin announces delay in starting RE-024 trial

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The NBIA Disorders Association posts the following announcement for informational purposes only. While the organization supports and encourages the discovery of treatments for NBIA individuals and willingly posts information concerning research studies (such as questionnaires and clinical trial enrollment), we do not endorse specific studies. Nor do we advise NBIA individuals or their families to take part in a particular study. Rather, we believe that those decisions are best made by affected individuals and/or their families, in collaboration with their doctors.

Retrophin recently informed the NBIA Disorders Association that the phase three trial of its PKAN drug, RE-024, is being delayed because of a manufacturing problem.

The trial’s placebo - often called a sugar pill because some patients will get it rather than the drug -“did not meet the stringent quality standards necessary for a clinical trial,” said Tricia Sterling, executive director of patient care at Retrophin.

The company had planned to start dosing patients by end of 2016 but now must correct the manufacturing issue so that the trial can proceed safely and generate high-quality clinical data, she said.

Delays in starting clinical trials are common for a variety of reasons.

“We are currently working diligently to remedy this manufacturing issue, and if all goes according to plan, we expect to begin dosing in the coming months,” Sterling said. “Please be assured that the delay is not related to problems with the RE-024 profile and is not expected to hinder the overall conduct of the clinical trial.”

The company hopes the drug ultimately will receive approval from the U.S. Food and Drug Administration, enabling Retrophin to deliver the first treatment option for PKAN patients.

Sterling said that, despite the delay in dosing patients, other aspects of the trial are proceeding as planned. Those steps include securing trial sites in the U.S., Canada and Europe. The company will provide information on the locations at, along with other trial details.

Retrophin said the most comprehensive information will be on the website it is launching as early as next week, Families may sign-up on to receive news releases. Those with questions about the study may contact the company at In addition, the NBIA Disorders Association will post updates on our website.



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