FDA sends once again Untitled Letter to Manufacturers of Regenerative Therapies

Recommendation

7/8 May 2024

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Regulatory Requirements and Practical Implementation

With the increasing importance of cellular and tissue-based products in the health care system, there is also an increasing number of companies that focus on this lucrative market and develop, manufacture and/or distribute products for treatment or therapy. In doing so, there seems to be a lack of awareness that one is entering the area of medicinal products which are subject to authorisation. (see also FDA admonishes Regenerative Therapies Manufacturer in Untitled Letter). This is also made clear in the letter from the FDA to the company Riverside Biologics. In this letter, the FDA writes that a cellular product derived from human umbilical cord is offered on the Riverside Biologics website. This is sold to consumers for the treatment of pain and various orthopaedic conditions. According to the information on the website, the product is administered by intra-articular or intramuscular injection.

The FDA notes that this is a product that qualifies as a human cellular, tissue, or cellular or tissue-derived product (HCT/P) as defined in 21 CFR 1271.3(d) and is hence subject to regulation. Accordingly, the appropriate premarket testing and investigation must be conducted if an exemption does not apply. In this regard, FDA says: "It appears that your company does not qualify for any of the exemptions in 21 CFR 1271.15 and that your above-mentioned product is intended for non-homologous uses. In addition, this product also does not appear to meet other criteria in 21 CFR 1271.10(a). Accordingly, it appears that the product would be regulated as a drug under the definition in section 201(g) of the FD&C Act [21 U.S.C. 321(g)] and as a biological product under the definition in section 351(i) of the PHS Act [42 U.S.C. 262(i)]." 

Accordingly, such a product would need to have a valid biologics licence in order to be marketed Such licences are only granted after it has been demonstrated that the product is safe, pure and effective. A development-stage application would be possible if the sponsor had filed an Investigational New Drug Application (IND) under FDA regulations.

The FDA refers in this context to the comprehensive regulations available on the FDA website.

FDA Recommendations

The FDA recommends manufacturers, health care professionals or the like who are uncertain about the regulatory status of their products to contact the FDA for a recommendation or decision regarding the classification of an HCT/P. For more information in this regard or on IND requirements for biological products, please see pages 24 and 25 of the guidance document titled "Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use".

The FDA sets a deadline of 30 days after receipt of the "Untitled Letter" to respond accordingly.