With the program’s seventh approval in the books, the FDA is illustrating just how speedy it can be with drug reviews that fall under its U.S. national priority pilot.
On Friday, two days after handing Partner Therapeutics a Commissioner’s National Priority Voucher (CNPV), the FDA gave the all-clear to the company’s bispecific antibody Bizengri as a second-line treatment for adults with advanced, unresectable or metastatic cholangiocarcinoma with an NRG1 gene fusion.
The green light in the rare bile duct cancer marks the third indication for Bizengri, which scooped up class-first accelerated nods in NRG1+ pancreatic adenocarcinoma and non-small cell lung cancer in late 2024. Partner Therapeutics licensed U.S. rights to the bispecific from its developer Merus in close conjunction with Bizengri’s first approval.
The new nod in cholangiocarcinoma, meanwhile, marks the latest in a series of swift approvals issued under the controversial CNPV pilot program, which the FDA debuted last June.
The program, which seeks to abbreviate review times to one to two months (down from 10 to 12 months for a traditional review) for applications that align with U.S. national priorities, has also bestowed approvals upon the likes of Novo Nordisk, Johnson & Johnson and Boehringer Ingelheim, among others.
“Patients with this ultra-rare type of cancer desperately need new treatment options,” embattled FDA Commissioner Marty Makary, M.D., said in a statement Friday.
He continued, “Through the national priority voucher pilot program, the FDA is accelerating therapies for rare diseases with unmet medical needs, reviewing applications in significantly shortened timelines.”
Prior to receiving the CNPV, Bizengri had secured FDA breakthrough therapy and orphan drug tags its cholangiocarcinoma indication, Partner noted in its own release late last week. The company announced on April 14 its filing with the FDA for the label expansion.
The new green light hinges on data from the medication’s midphase eNRGy trial, in which 22 patients were enrolled and 19 were evaluable for efficacy, according to the company. In the study, Bizengri helped 36.8% of those cholangiocarcinoma patients achieve a response, with the duration of that response lasting between 2.8 months to 12.9 months.
“The results demonstrate meaningful tumor responses, durable benefit, and a favorable tolerability profile—and we are grateful that the FDA’s Commissioner’s National Priority Voucher pilot program greatly reduced the review time, helping bring this treatment to patients more quickly,” Pritesh Gandhi, chief development officer at Partner, said in a statement.
Cholangiocarcinoma is a rare and aggressive bile duct cancer with an all-stage five-year overall survival rate of less than 15%, according to Partner. The NRG1 gene fusions covered in Bizengri’s new label crop up in fewer than 1% of cholangiocarcinoma cases and typically occur in younger adult patients who are otherwise driver-negative, leaving them without approved targeted therapy options, the company explained.
While seven drugs have now passed through the CNPV pilot, the program has attracted its share of controversy too. Lawmakers last year shared “deep concerns” that the program could be vulnerable to corruption and favors for drugmakers and allies in the Trump administration’s good graces. Concerns have also mounted that the process could lead to rushed reviews and undermine the FDA’s regulatory discipline.
The FDA has a public hearing scheduled for June 4 to receive input on the program and has specifically singled out issues for public comment around eligibility criteria, the selection processes, sponsor responsibilities, pre-submission requirements, FDA review procedures and other aspects of the program’s implementation.
