Regulators opening new pathways to register chemically-synthesized proteins as Biosimilars

Biological medicines, and anticancer biologicals in particular, represent a growing burden for healthcare budgets. As a result of the loss of exclusivity rights on original biologicals, biosimilar medicines have been allowed to enter the market to offer cost-effective treatment options.
Through the Biologics Price Competition and Innovation Act (BPCI), the US Congress created an abbreviated approval pathway as a way to provide the public with greater access to biological products that are effective and safe.

In 2017, the US FDA issued proposed guidelines making chemically-synthesized peptides up to 40 amino acids eligible to an Abbreviated New Drug Application (ANDA) in reference to a previously approved peptide of rDNA origin. Two essential conditions were set:
1. The “proposed generic synthetic peptide” should have the “same” active ingredient than the previously approved peptide of rDNA origin.
2. The acceptability of the “synthetic peptide” intended to be a “duplicate” of a previously approved peptide of rDNA-origin, would largely depend on its impurity profile as compared to the impurity profile for the peptide rDNA origin.

As a further step in the implementation of the BPCI Act, the FDA redefined the word “protein” as “any alpha amino acid polymer with a specifc sequence that is greater than 40 amino acids” and removed from the previously definition of a biological product “protein (except any chemically synthesized polypeptide).” These changes were enacted in March 2020.

This move by FDA opens new pathways for manufacturers to bring biosimilars, including chemically-synthesized proteins, to market.
We strongly encourage our partners to reach out to FDA early in their development program of Biobetters to discuss issues related to product classification and the appropriate pathway for a marketing application.