Microbiome-based therapeutics: FDA approvals

Author: Luke Springall, Senior Market Analyst, Hanson Wade Intelligence

A growing field

The microbiome-based therapeutics field has exploded in recent years due to an increasing number of studies showing associations between the microbiota composition in the gut and illness, from depression to neurodegeneration. To explore this further, we have examined the data in Beacon. Beacon has identified 738 microbiome programs, with 358 in preclinical development and 317 clinically active. However, the field is still relatively new, and most clinical trials are in Phase I/II. 

Therapeutic Diversity

This area has an incredible range of therapies available, including prebiotics, live biotherapeutics, bacteriophage cocktails, and faecal matter transplants, which deliver all microbes and gut nutrients. The hard work and ever-growing data were noticed, and at the end of 2022, the field achieved a massive milestone with approvals from the FDA. 

Two Q4 Approvals

Two microbiome products were approved for restoration of gut microbiota in the treatment of recurrent Clostridioides difficile infection, which is associated with diarrhea and continues to demonstrate significant global unmet medical need. The Australian-based BiomeBank developed a whole microbiota transplant, BIOMICTRA, the first microbiome product that was approved on 9th November 2022. This was swiftly followed by REBYOTA from Ferring Pharmaceuticals on 30th November with their live biotherapeutic. These two products are neatly illustrative of the current drug landscape. In 2022 alone, 288 and 199 assets were announced for live biotherapeutics and whole ecosystem-derived products, respectively. However, the field is more expansive than these two areas, and eye-catching programs continue to grow. As of 13th February 2023, our team at Beacon has identified 526 trials within the microbiome therapeutics space, with AAGR since 2012 of 25% and 130 trials expected to be initiated in 2023. 

Future approvals

The field is at an exciting moment with two recent FDA approvals, and an increasing number of trials initiated. The scope of disease states targeted is vast and comparable to the diversity in niche therapeutic approaches. Demonstrating effectiveness and safety will continue to be the highest priority. There are reasonable concerns with the purity and stability of the products, especially live biotherapeutics, for treatment efficacy. Further study of the intestinal microbiome and patient responses is also required, as some treatments have been shown to be transient, with patients returning to baseline after a few weeks. The field is in its early stages, and with Beacon showing 21 programs in Phase III, the next approval will not be too far away.