AcelRx Receives IDE Approval for Niyad and Advances to a Single Registration Study

AcelRx Pharmaceuticals, a specialty pharmaceutical company focused on innovative therapies, has received Investigational Device Exemption (IDE) approval from the U.S. Food and Drug Administration (FDA) for its product candidate, Niyad™. This milestone allows AcelRx to move forward with a pivotal study to evaluate the safety and efficacy of Niyad as a regional anticoagulant for the extracorporeal circuit.

Niyad, a lyophilized formulation of nafamostat, will be evaluated in a registrational study involving 166 adult patients undergoing renal replacement therapy (RRT) who cannot tolerate heparin or are at risk for bleeding. The study, expected to begin in the fourth quarter of 2023, will be conducted at approximately 10 hospital intensive care units in the United States.

If approved, Niyad would become the first and only FDA-approved regional anticoagulant for the extracorporeal circuit in the U.S., offering a groundbreaking treatment option for patients in need. The study’s primary endpoint will be mean post-filter activated clotting time (ACT) over the first 24 hours compared to a placebo, with key secondary endpoints including filter lifespan, number of filter changes, number of transfusions, and dialysis efficacy.

Vince Angotti, CEO of AcelRx, expressed gratitude for the FDA’s approval and highlighted the potential of Niyad as a paradigm-shifting product candidate for the medically supervised setting. The company aims to start the study later this year, with top-line data expected by mid-2024 and a premarket approval application (PMA) submission planned for the second half of 2024.

Dr. Pamela P. Palmer, Chief Medical Officer and Co-Founder of AcelRx, emphasized the positive interactions with the FDA and the acceptance of a straightforward registrational trial design for Niyad. She further noted the commitment to advancing Niyad as a regional anticoagulant during renal replacement therapy, addressing the limitations of current options and ensuring patients receive care according to international standards.

AcelRx Pharmaceuticals is dedicated to developing and commercializing innovative therapies for use in medically supervised settings. In addition to Niyad, the company is also working on other product candidates, including Fedsyra™ and PFS-02.

For more information about AcelRx Pharmaceuticals, please visit www.acelrx.com.