Microbiome therapeutics has long been the playground of small biotech companies. But as recent investments have shown, big pharma has been quite interested in entering the game. For example, AstraZeneca invested $20 million in Seres Therapeutics to work on microbiome medicines in immuno-oncology while AbbVie has elected to collaborate with Synlogic to target the inflammatory bowel disease market. As pharma get more implicated in the microbiome space, their involvement becomes key to the development of a regulatory framework.
In the US, the current regulatory pathway for microbiome therapeutics is still being defined. While three drugs are going through phase III trials: two have done so by using an orphan drug status (SER-109 by Seres Therapeutics and RBX-2660 by Rebiotix which are specifically indicated for the treatment of C. difficile infection) while the third, RP-G28 (Ritter Pharmaceuticals) is in trials for the treatment of lactose intolerance. The orphan drug regulatory strategy, while enabling product approval, has a significant downside for even when these microbiome therapeutics are approved, they will achieve an orphan drug status. This means they will be available uniquely for a specific indication, and end up being priced accordingly. Developing more encompassing regulatory pathways for microbiome therapeutics is necessary going forward, but our understanding of the microbiome is limited, which has complicated the development of a distinct regulatory framework. Relying on innovative data points (such as real-word data) to demonstrate value might be a valuable strategy to reduce regulatory agency anxiety.iv
Its important to note that regulatory bodies are usually quite willing to work early with innovators to develop standardized regulatory frameworks, and usually look to industry to assist in the preparation of practical real-world frameworks. For big pharma, this will mean developing frameworks for two key requirements. First, a framework to work on the characterization of microbiome products and second, frameworks for well-designed clinical studies with defined endpoints.
