With the program’s seventh approval, FDA is demonstrating how quickly it can review drugs that qualify for national priority testing in the United States.
On Friday, two days after handing Partner Therapeutics the Secretary’s National Preference Voucher (CNPV), the FDA fully cleared the company’s bispecific antibody Bizengri as a second-line treatment for adults with advanced unresectable or metastatic cholangiocarcinoma harboring the NRG1 gene fusion.
The green light in rare bile duct cancer marks Bizengri’s third indication, with first-in-class accelerated approvals in NRG1+ pancreatic adenocarcinoma and non-small cell lung cancer in late 2024. Partner Therapeutics worked closely with Bizengri’s initial approval to license the U.S. rights for the bispecific disease from developer Merus.
Meanwhile, the new approval for bile duct cancer is the latest in a series of expedited approvals issued under the controversial CNPV pilot program that the FDA debuted last June.
The program aims to reduce the review time for applications aligned with U.S. national priorities to one to two months (compared to 10 to 12 months for traditional reviews) and has also granted approvals to Novo Nordisk, Johnson & Johnson, Boehringer Ingelheim, and others.
“Patients with this ultra-rare type of cancer desperately need new treatment options,” embattled FDA Commissioner Dr. Marty McCulley said in a statement Friday.
He added: “Through the National Priority Voucher Trials Program, FDA is accelerating treatments for rare diseases with unmet medical need and reviewing applications on a significantly accelerated timeline.”
Prior to receiving CNPV, Vizengri had secured the FDA’s breakthrough therapy and an orphan drug tag for the bile duct cancer indication, the partners said in their own release late last week. The company announced on April 14 that it has applied to the FDA for label expansion.
The company said a new green light is dependent on data from the drug’s mid-term eNRGy trial. The trial enrolled 22 patients, and 19 were able to evaluate efficacy. In this study, Vizengri helped 36.8% of cholangiocarcinoma patients achieve a response, with response durations lasting between 2.8 and 12.9 months.
“These results demonstrate meaningful tumor responses, durable benefit, and a favorable tolerability profile, and we are grateful that the FDA Commissioner’s National Priority Coupon Trials Program has significantly shortened review times and brought this therapy to patients more quickly,” Pritesh Gandhi, the partners’ chief development officer, said in a statement.
According to Partner, cholangiocarcinoma is a rare and aggressive form of bile duct cancer with a five-year overall survival rate of less than 15% for all stages. The company explained that the NRG1 gene fusion covered by Vizengri’s new label occurs in less than 1% of bile duct cancer cases, typically in young adult patients who are driver negative, a condition for which there are no approved targeted therapy options.
Seven drugs are currently going through the CNPV pilot, a program that has generated similar controversy. Lawmakers last year shared “deep concerns” that the program could be vulnerable to corruption and favoritism to drug companies and allies favored by the Trump administration. There are also growing concerns that the process could lead to rushed reviews and undermine the FDA’s regulatory discipline.
FDA has scheduled a public hearing on June 4 to obtain input on the program and has identified issues for public comment regarding, among other things, eligibility criteria, selection processes, sponsor responsibilities, pre-submission requirements, FDA review procedures, and other aspects of program implementation.
