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. 2024 Aug 16;230(2):505-513.
doi: 10.1093/infdis/jiae146.

Regulatory Considerations in the Approval of Rezafungin (Rezzayo) for the Treatment of Candidemia and Invasive Candidiasis in Adults

Heidi L Smith  1 , Timothy J Bensman  2 , Shrimant Mishra  1 , Xianbin Li  3 , Cheryl A Dixon  3 , Jalal Sheikh  1 , Owen G McMaster  4 , Abhay Joshi  2 , Daniel B Rubin  3 , Avery Goodwin  1 , Terry J Miller  4 , Zhixia Y Danielsen  2 , Irum Syed  5 , Sunita J Shukla  6 , Dmitri Iarikov  1 , Peter W Kim  1 , John J Farley  6
Affiliations

Regulatory Considerations in the Approval of Rezafungin (Rezzayo) for the Treatment of Candidemia and Invasive Candidiasis in Adults

Heidi L Smith et al. J Infect Dis. .

Abstract

On 22 March 2023, the FDA approved rezafungin (Rezzayo) for the treatment of candidemia and invasive candidiasis in adults with limited or no alternative treatment options. Rezafungin is an echinocandin that supports weekly dosing, enabling outpatient parenteral treatment that potentially avoids the need for a central venous catheter. Approval of rezafungin was based on a single adequate and well-controlled phase 3 study designed with a day 30 all-cause mortality primary end point and 20% noninferiority margin, which demonstrated that rezafungin is noninferior to the comparator echinocandin. Nonclinical studies of rezafungin in nonhuman primates identified a neurotoxicity safety signal; however, rezafungin's safety profile in the completed clinical studies was similar to other Food and Drug Administration-approved echinocandins. Here we describe the rationale for this approval and important considerations during the review process for a flexible development program intended to expedite the availability of antimicrobial therapies to treat serious infections in patients with limited treatment options. Clinical Trials Registration . NCT02734862 and NCT03667690.

Keywords: candidemia; drug approval; echinocandins; invasive candidiasis; rezafungin.

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Conflict of interest statement

Potential conflicts of interest. All authors: no reported conflicts. All authors have submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Conflicts that the editors consider relevant to the content of the manuscript have been disclosed.

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