Generon moving ahead with Phase 3 trial following SPA
- A Chinese biotech has squared away the design for a late-stage study of its lead candidate, providing a "clear path forward" as it works to gain the Food and Drug Administration's approval, according to a Wednesday announcement.
- The randomized, open-label trial evaluates Generon Corp. Ltd.'s F-627, also called Binueuta, as a treatment for cancer patients with low white blood cell counts caused by chemotherapy. The trial will specifically enroll breast cancer patients who are receiving chemotherapy, and give them either Generon's drug or a standard dose of a comparator — in this case, Amgen Inc.'s Neulasta.
- Generon devised the study design through a Special Protocol Assessment (SPA), a procedure that drugmakers use to get FDA feedback on clinical investigations. "The SPA provides a binding agreement with the FDA’s review division that the Phase 3 trial can adequately address objectives in support of a U.S. regulatory submission for approval of F-627," the company said.
Neutropenia drugs have become a valuable addition to pharmas' arsenals. Amgen's Neulasta (pegfilgrastim), an approved treatment for decreasing incidence of infection spawned from febrile neutropenia, brought in more than $1 billion for the big biotech during the second quarter.
Sensing the potential returns, companies have made a push to develop more drugs for the therapeutic area. Mylan NV and Biocon are working on their own biosimilar version of Neulasta, though they've been stymied by FDA delays. In any case, the neutropenia market doesn't appear to be slowing down — last year the chemotherapy-induced neutropenia market reached $3.67 billion, according to a recent report.
SPAs, meanwhile, have become a vital tool for pharmaceutical companies looking for clarity on whether their research efforts hold water. Last year, the FDA updated guidance from 2002, indicating that there are select types of studies for which a drugmaker can request an SPA. They include animal carcinogenicity investigations, drug substance and drug product stability research, and trials meant to show biosimilarity or interchangeability.
Yet, while SPAs are a useful source of regulatory insight, they're no catch all. The FDA can reject an SPA request. And even if the agency does grant one, there's not guarantee the corresponding drug's approval filing will be accepted.
"Although the goal of an SPA is to reach concurrence on the adequacy of protocol elements intended to support a statutory finding of safety and efficacy, an SPA agreement with FDA does not imply that FDA has reviewed or concurs with each detail of the protocol," the agency wrote in a guidance document published in 2016.
"For example, an SPA agreement for a protocol might communicate to the sponsor that FDA agrees with the proposed primary endpoint or the sample size estimate, but might not include a detailed review of the case report form; it might address the adequacy of and final timing of a radiographic procedure used to measure the primary endpoint, but might not comment on the use of three versus four interim radiographs."
For Generon, however, the SPA is a step in the right direction.
"Obtaining the FDA’s agreement on the F-627 Phase 3 trial design, endpoints, patient population and statistical approach is a significant accomplishment," David Lacey, chairman of Generon’s scientific advisory board, said in an Oct. 10 statement. "Collaborative discussions with the FDA led to a positive outcome with this SPA that reduces regulatory risk for this product."
- Generon Statement
- Food and Drug Administration Guidance for Industry — Special Protocol Assessment
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