December 26, 2018
The FDA recently vowed to crackdown on unsubstantiated and poor quality stem cell therapies in 2019. Biologic-based products and other emerging medical technologies are at the center of a debate over the best regulatory pathways to ensure patient safety.
Carmell Therapeutics' President and CEO Randy Hubbell spoke with Becker's Spine Review about regulatory pathways for biologic products and the company's plans for the spine space.
Question: Why is Carmell opting for the FDA's Biologics License Applications approval route?
Randy Hubbell: There are two pathways to regulate human cell and tissue products: the FDA's 351 pathway is for cell- or tissue-based products that are more than minimally manipulated and the FDA's 361 pathway is for biologic-based products used in a homologous- and minimally-manipulated way.
There are big differences between the two pathways, and the 361 pathway is somewhat controversial. Products regulated by the 361 pathway are not required to follow the FDA's submission pathway, whereas the 351 regulatory pathway — the BLA pathway — is the most robust.
Carmell is conducting two phase 3 clinical studies of our Bone Healing Accelerant product simultaneously, since BLA biologics require two reproducible studies for approval. This is a higher bar and standard for both 361’s and medical devices — a PMA, for instance, the highest standard for medical device approval and only 5 percent of total medical devices approved, only requires one pivotal study for approval.
We believe that the 351 regulatory pathway will create substantially more value for the company, as it is a regulatory pathway demonstrating the product is made in a safe and effective way and is clearly superior to standard of care.
Question: How does the debate over regulatory pathways impact the industry more broadly?
RH: The surgical, orthopedic and wound healing space is used to seeing products introduced through the FDA 510(k) or 361 pathways. That whole space is lacking in robust regulatory approvals and clinical data to support the claims being made, and hospitals are scrutinizing technologies without robust regulatory approval and data. They're looking at the benefit to the patient, what the product is going to replace and the cost benefit to the hospital. Surgeons can no longer justify the acquisition of new technologies based solely on physician preferences, so requiring a more rigorous regulatory approval process can control costs.
Question: What is Carmell planning for the spine space?
RH: The second indication of our first product, the Bone Healing Accelerant for severe long bone fractures, is for spinal fusion. We're studying the ability to mix our technology with demineralized bone matrix products, since it's the standard of care to fill the intervertebral gap to facilitate bone growth. In preclinical models, we've seen bone grow faster, in a higher-volume and at a greater density. Now we're laddering up to a larger preclinical model on a path to a label expansion for our first product.
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