Entries from November 2009 ↓

Will Fibrocell Sciences be the 1st Regenerative Medicine Product Approved by the FDA in over 12 Years?

I had a chance to attend part of the October 09, 2009 FDA Cellular, Tissue and Gene Therapies Advisory Committee meeting to discuss the biologic license application (BLA) for Fibrocell Science’s autologous fibroblast product intended to treat nasolabial fold wrinkles.

Don’t be surprised if you have never heard of Fibrocell Science, Inc. Prior to filling for Chapter 11 bankruptcy and reorganizing, they were known as Isolagen, Inc.

Based on the discussion at the meeting and in the FDA briefing document, it appears Fibrocell Science’s clinical data is adequate to demonstrate clinical efficacy.  Despite the challenges inherent in manufacturing of autologous products and given the relative ease of culturing fibroblasts, it appears there were no major manufacturing issues identified by the FDA.  The presentation given by Fibrocell Science at the meeting an other supporting information can be found at a separate link. I was not able to stay for the entire meeting, but in general the members of the advisory committee seemed satisfied with the clinical efficacy data.  However, there was a lengthy discussion about the overall evidence for clinical safety, due in part to question #1, posed by the FDA to the committee (note: “IT” refers to Isolagen Therapy, since at the time of the BLA submission in May 2009, the company was still going by the name of Isolagen:

Tumorigenicity: If approved, IT will be the first cellular product for this indication, and the first fibroblast product that is an injectable cell suspension. Uncontrolled cell growth and/or tumor formation could be potential risks of cultured fibroblasts due to their proliferative nature. In addition, there is a theoretical risk of the post-auricular biopsy transferring abnormal or malignant cells that may not be detected in the quality controls of product manufacturing. Long term follow-up data are limited. One case of basal cell cancer occurred near the site of injection; however, the relationship of IT to this case cannot be assessed.

As a result, the committee voted 11 (yes) to 3 (no) that the autologous fibroblast product demonstrated efficacy for the proposed clinical indication, but 6 (yes) to 8 (no) that the clinical data demonstrated safety for the proposed indication.  This is quite interesting since in order  for FDA to issue a BLA, the product must be demonstrated to be both safe and effective.  Usually efficacy is the primary issue in a BLA, at least for cell-based therapies.  From what I understand from the discussion at the advisory committee meeting there was no pattern, or other clear evidence, that the autologous fibroblast product presents a safety risk to patients.  However, the FDA must ensure the potential benefits to patients outweigh the risks.  Given the non-life threatening indication (wrinkles), it is prudent for the FDA to be cautious.  Despite this, I am cautiously optimistic that a BLA will issued for this product, albeit with post-approval (Phase IV) commitments to further study the short and long term safety profile of the product.  If you were at the meeting or have other insights regarding this product, please share them in the comments.