NBIAcure Prepares to Submit for FDA Approval of CoA-Z Compound
March 2024
By Amber Denton
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| Dr. Penelope Hogarth and Dr. Susan Hayflick, NBIAcure Oregon Health & Science University, Portland, Oregon |
NBIAcure, a research team led by Dr. Susan Hayflick and Dr. Penny Hogarth and based at Oregon Health & Science University (OHSU), has released a significant update to the Pantothenate Kinase-Associated Neurodegeneration (PKAN) community regarding the status of CoA-Z, a vitamin intermediate. In January, the team announced their decision to approach the U.S. Food and Drug Administration (FDA) based on the strength of gathered data rather than pursue additional studies.
The FDA will evaluate data from the clinical trial to assess the safety and tolerability of CoA-Z and its effect in altering a biological marker of PKAN in the blood. Trial results indicate that CoA-Z altered the blood biomarker in a dose-dependent manner, with higher doses yielding a greater response.
A critical outcome of the trial is demonstrating CoA-Z's safety. This involves establishing that possible side effects occur with the same frequency, whether a participant was on a placebo or one of three doses.
The initial six months of the trial were conducted in a double-blind, placebo-controlled manner to meet the most rigorous scientific requirements. This approach demonstrates to the FDA that the research was done with the greatest integrity to protect against potential bias. Analysis of this phase confirmed CoA-Z's safety and tolerability across all tested doses.
The team is finalizing the report for this phase alongside other essential information, including the manufacturing process, stability under varied conditions, and effects on animal models. The target is to submit a draft application to the FDA by the end of March, a goal that remains on track.
Although the exact timeline for the FDA's review of the draft remains uncertain, the agency has suggested that conducting a preliminary assessment before final submission could minimize future delays. The team will advocate for expedited review while valuing the time and attention given to the review process. Meanwhile, the focus will shift to analyzing data from the trial's open-label portion, where all participants received the same dose of CoA-Z for inclusion in the final submission.
In February, Dr. Penelope Hogarth, part of the NBIAcure team at OHSU, presented the clinical trial results at the 8th International Symposium on Pediatric Movement Disorders in Barcelona, Spain. Numerous PKAN specialists attended the symposium and expressed excitement about the findings.
NBIAcure and the Spoonbill Foundation partners remain hopeful that the presented data will suffice to convince the FDA that additional studies are unnecessary. The team extends their gratitude to the PKAN community for their support and encouragement throughout this process.
