ADC Reviews

FDA Orphan Drug Designation and Fast Track Designation for rezafungin


On September 20, Cidara/Melinta Therapeutics jointly announced that the FDA has accepted the New Drug Application (NDA) for rezafungin (rezafungin) for the treatment of candidemia and invasive candidiasis, and granted it priority review status, PDUFA The date is March 22, 2023. Previously, the FDA has certified Rezafungin as a Qualified Infectious Disease Product (QIDP), as well as Orphan Drug Designation and Fast Track designation.

Invasive candidiasis (IC) is a severe and life-threatening systemic Candida infection that can infect the bloodstream and/or deep/visceral tissues. Caspofungin (administered once a day) is the current standard therapy, but the mortality rate of IC patients is still as high as 40%, and new treatment options are urgently needed.

Echinocandins are a class of antifungal drugs that noncompetitively inhibit β-1,3-glucose synthase, thereby disrupting the integrity of the fungal cell wall. Razafungin is a novel echinocandin with a long half-life that enables once-weekly dosing. On August 22, the marketing application of Rezafungin for the treatment of adult patients with invasive candidiasis was accepted by the European Medicines Agency (EMA).

The marketing application is based on positive data from the Phase III clinical trial ReSTORE and the Phase II clinical trial endeavor. The two trials were designed to evaluate the efficacy and safety of razafungin in patients with candidemia and/or invasive candidiasis, with the primary endpoint being all-cause mortality at day 30.

Results showed that all-cause mortality was statistically non-inferior to caspofungin (once-daily dosing) in the razafungin group (once-weekly dosing), meeting the primary endpoint.

At the same time, razafungin and caspofungin have a similar safety profile and are well tolerated.

On July 27, Cidara reached a cooperation with Melinta, and Melinta obtained the rights to commercialize in the United States. Under the terms of the agreement, Cidara receives a $30 million down payment and is eligible to receive $60 million in regulatory milestones and up to $370 million in commercial milestone payments, as well as tiered royalties.

Jeff Stein, president and chief executive officer of Cidara Therapeutics, said: “This listing is an important step in the fight against difficult and often fatal candidiasis and invasive candidiasis for patients, and is an important step in Cidara’s razafungin development program. Milestone. Data generated from our Phase II and Phase III trials suggest that razafungin could change the current standard of care for the treatment of invasive Candida infections, and we are pleased that razafungin may be the first approval in more than a decade New drugs for this indication.”