After accepting Sanofi’s application for an expansion of its Type 1 diabetes treatment Tzield and putting it in the fast lane for a potential approval under a controversial new pathway, the FDA has signed off on the expansion, albeit nearly two months later than the original target date.
The accelerated nod will allow patients ages 8 to 17 who have been recently diagnosed with stage 3 Type 1 diabetes to use Tzield to delay the decline in endogenous insulin production. The monoclonal antibody was first approved four years ago by the FDA to delay the onset of stage 3 diabetes in those with stage 2 disease.
Sanofi’s expansion bid was accepted under former FDA chief Marty Makary’s Commissioner’s National Priority Voucher (CNPV) program, with the agency originally eyeing an April 21 decision.
After the date came and went without a complete response letter (CRL), Stat reported that the acting director of the FDA’s Center for Drug Evaluation and Research (CDER) at the time, Tracy Beth Hoeg, M.D., Ph.D., overturned a staff decision to endorse the expansion and that Sanofi had asked that the application be pulled from the CNPV program.
In the span of a few days last month, Makary resigned and Hoeg was fired, with Makary’s departure raising questions about the status of the CNPV program. Since it was established, the initiative was criticized as a potential tool for government officials to curry favor and reward political allies in the biopharma industry.
Of the first wave of nine drugmakers who got CNPVs, Sanofi was among those who did not apply. The FDA nominated the company to receive one because its application addressed an unmet need, Sanofi said upon accepting the nomination in October of last year.
On Monday, a Sanofi spokesperson said that Tzield was no longer part of the CNPV program.
“The FDA has informed us that it will no longer be conducting an advisory committee meeting,” the spokesperson added in an email. “During the continued review process, all outstanding questions were clarified, enabling the FDA to grant the accelerated approval.”
The nod was backed by data from the Protect phase 3 study of 328 participants ages 8 to 17 who had been diagnosed with stage 3 diabetes within the last six weeks. Tzield slowed the decline in C-peptide levels compared to placebo. C-peptide is a short chain of amino acids released into the bloodstream in equal amounts to insulin production.
“We now have a novel therapy that targets the autoimmune and progressive nature of stage 3 type 1 diabetes,” Aaron J. Kowalski, Ph.D., the CEO of Breakthrough T1D, said in a statement. “Approximately 64,000 people are diagnosed with T1D every year. We are excited that the approval of Tzield in this indication provides a treatment option for certain patients diagnosed in stage 3 T1D.”
Sanofi added that Tzield is not effective as a disease-modifying therapy in non-autoimmune dysglycemic conditions.
Sanofi currently is enrolling patients in a confirmatory phase 3 study, which is designed to demonstrate the clinical benefit of Tzield in the indication. The Protect trial was based on a surrogate C-peptide endpoint deemed likely to show clinical benefit.
The nod comes on top of an FDA expansion two months ago for Tzield to treat patients age 1 and older. Last year, Sanofi reported sales of Tzield at 68 million euros ($77 million).