FDA Guides the Way to Biosimilars in the US

Has the long-awaited guidance answered all of the industry's questions?
Mar 02, 2012


Angie Drakulich
Almost two years to the date after President Obama signed the Affordable Care Act, introducing a regulatory pathway for biosimilar approval in the United States, FDA has released draft guidance for industry about how to get such a product into the marketplace.

Industry has been at a pivotal standstill since the Biologics Price Competition and Innovation Act (BPCI Act) was passed in 2010, as part of the government's overall healthcare act, but biologics developers and manufacturers seem ready to pounce. According to a press briefing given by FDA's Rachel Sherman in early February, there have been 35 pre-investigational new drug (IND) meeting requests for proposed biosimilars to 11 reference products, 21 pre-IND sponsor meetings held, and 9 INDs received. Sherman is the associate director for Medical Policy within FDA's Center for Drug Evaluation and Research. With the doors to FDA's biosimilar review desk widened, those numbers are likely to skyrocket in the coming months.

The draft guidance—three documents to be exact—includes Scientific Considerations in Demonstrating Biosimilarity to a Reference Product; Quality Considerations in Demonstrating Biosimilarity to a Reference Protein Product; and Biosimilars: Questions and Answers Regarding Implementation of the Biologics Price Competition and Innovation Act of 2009. FDA seems to have gone out of its way to anticipate and respond to key concerns in the documents, the first being whether animal and clinical data from a non-US licensed comparator drug can be used to demonstrate biosimilarity. The answer is yes, according to the Scientific draft guidance, although justification and adequate bridging data to a US-licensed reference product will be required in these cases.

Another question addressed is which studies a sponsor needs to perform and submit as part of its 351(k) application—the route to be taken for biosimilar approval. The BPCI Act set the stage for these requirements, and the draft Scientific guidance states that companies must include "analytical studies that demonstrate that the biological product is highly similar to the reference product notwithstanding minor differences in clinically inactive components; animal studies (including the assessment of toxicity); and a clinical study or studies (including the assessment of immunogenicity and pharmacokinetics or pharmacodynamics) that are sufficient to demonstrate safety, purity, and potency in one or more appropriate conditions of use for which the reference product is licensed and intended to be used and for which licensure is sought for the biological product."