Together with new guidance regarding the potential regulatory pathways for gene therapies, the FDA announced on Monday, August 28, 2017, that it will be taking action against companies providing unapproved stem cell treatments. This new guidance, combined with the threat of enforcement actions, provides the regenerative medicine industry and its partners with certain direction regarding the differences between pioneering stem cell therapies for which regulatory pathways are being crafted and unapproved treatments, which are likely to trigger scrutiny by the FDA.

Overview of enforcement actions

On August 24, the FDA issued a warning letter to a Florida clinic that recovered a patient's own adipose tissues, processed those tissues into stromal vascular fraction (SVF) — i.e., adult stem cells derived from the patient's own fat — and then administered the SVF to the patient for treatment of any one of several conditions, including Parkinson's disease, amyotrophic lateral sclerosis (ALS) and chronic obstructive pulmonary disease (COPD).

The FDA rejected the clinic's arguments that

  1. The SVF qualified as human cells, tissues, and cellular and tissue-based products (HCT/Ps), which may be manufactured and sold without pre-market authorization; and
  2. The procedure qualified for the "same surgical procedure" exception at 21 CFR §1271.15(b).

Instead, the FDA took the position that the post-recovery processing of the adipose tissues takes the procedure outside of both the definition of an HCT/P and the same-surgical-procedure exception. The agency further noted that use of SVF for conditions such as Parkinson's disease, ALS and COPD do not meet the homologous use requirement of 21 CFR §1271.10. The agency also announced an action against a separate stem cell therapy clinic in California.

In light of these developments, practitioners and clinics who are

  1. Engaged in SVF-enhanced adipose transplantation or similar treatments involving the enhancement or processing of cells and tissues for autologous use and
  2. Relying on either the premarket treatment afforded HCT/Ps or the same-surgical-procedure exception should revisit the regulatory pathway for their products or services.

Guidance for regenerative medicine companies

While announcing these regulatory and enforcement actions, the FDA also sought to reassure the regenerative medicine industry of the agency's dedication to promoting this field of science. FDA Commissioner Scott Gottlieb wrote that the agency plans to "advance a comprehensive policy framework that will more clearly describe the rules of the road for this new field." Manifesting the agency's willingness to work with industry, Dr. Gottlieb noted that while "[m]any of the individualized treatments fall clearly outside the FDA's pre-market requirements…[f]or those that currently fall across the line and are subject to the FDA's existing pre-market review, we want to make sure the process for gaining FDA approval is efficient. We want to facilitate innovation." Thus, while signaling its intent to take action against entities it believes are "targeting vulnerable patients," the FDA indicated that it "will give current product developers a very reasonable period of time to interact with the FDA in order to determine if they need to submit an application for marketing authorization and to come into the agency and work on a path towards approval."

The FDA also reaffirmed its commitment to fully implementing the regenerative medicine advanced therapy (RMAT) designation process. The designation is given to cell therapies, therapeutic tissue engineering products, human cell and tissue products, or any combination product using such therapies or products intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and for which preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition. See Section 3033 of the 21st Century Cures Act.

These are important developments for stakeholders across the regenerative medicine industry, including:

  • Companies researching or involved with providing stem cell therapies
  • Partners of these companies, whether suppliers, distributors, or other co-venturers
  • Investors in these companies, particularly, but not limited to, those involved in stem cell therapies
  • Insurance companies providing representations and warranties (R&W) insurance for transactions involving regenerative medicine companies