Entries Tagged 'OCTGT' ↓

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.


IXA Statement on Xenotransplants for Type 1 Diabetes

For those of you following developments in the field of xenotransplantation, the International Xenotransplantation Association (IXA) has recently developed a proposed Consensus Statement on Conditions for Undertaking Clinical Trials of Porcine Islet Products in Type 1 Diabetes.

In my quick read of the statement it appears to be a comprehensive, well thought out document that encompasses ethical and public health considerations as well manufacturing, preclinical and clinical considerations.    The statement was drafted by an number of thought leaders in the field, such as Bernhard Hering, Greg Korbutt, Clive Patience, Harold Vanderpool and Henk-Jan Schuurman.  It also appears that the FDA provided some informal feedback on the statement, since Keith Wonnacott from FDA is mentioned in the acknowledgments.

I get the general impression that many assume the field of xenotransplantation is “dead,” because of  the challenges the field has faced due to public health concerns such as porcine endogenous retrovirus (PERV).  However, it appears the field has now been able to largely address those concerns.

Since it is clear that there continues to be  large unmet medical needs that are not yet met by any existing medical products, there has been a small but dedicated group of individuals who are attempting to determine if animal-derived cells and tissues can help.  In my view, xenotransplantation, as a cell replacement therapy,  fits within the scope of regenerative medicine.

I think this consensus statement from IXA represents one example of how complex areas of medical research, which raise an number of important issues in the minds of the public, can progress in a responsible manner.  Let me know your thoughts in the comments.


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Cautionary Tale: Tumors Caused by Neural Stem Cells

I usually avoid commenting on current events occurring in the field of regenerative medicine, primarily because I can’t keep up!  In this case,  I believe the recent publication by Amariglio et al, has FDA regulatory implications for the clinical development of therapies based on stem cells.  The under-lined portion of the title of the publication says it all:

Donor-Derived Brain Tumor Following Neural Stem Cell Transplantation in an Ataxia Telangiectasia Patient”

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My 2008 Presentations & Articles Relevant to Regenerative Medicine

2008 was an extremely busy year for me, as I am sure it was for you as well. The net result is that I have been slow to post updates and thoughts regarding translation of regenerative medicine products to the clinic. In the ‘better late than never” category, I thought I would share some of the presentations and relevant articles I have written over the past year.

Links are provided for most of the presentations and articles I have generated over the past year.

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13 Tips for Successful FDA Meetings

There are many reasons for meeting with the FDA during development of a regenerative medicine product. Some regenerative medicine products are more novel than others, meaning they are so unique they have never been studied in human clinical trials. Others may not be first of its kind, but may be used in novel clinical applications or be manufactured in new ways in comparison to competitor products. The nuts and bolts of preparing to meet with the FDA will be covered in a separate posting.

There are a number of opportunities for informal and formal communications with the FDA. For the purpose of this post, I am assuming you have requested a formal meeting with the FDA, perhaps to discuss the filing of an investigational new drug (IND) application to initiate human clinical studies.

Usually, to reach this point you will have expended a significant amounts of time and money. Therefore you don’t want to blow it by not being prepared to meet effectively with the FDA. Below is my list of tips to help the process go more smoothly:

  1. Remember, The FDA Wants You to Succeed
  2. Leave the “Dog & Pony” Show at Home
  3. Safety is the FDA’s Primary Focus
  4. Provide Clarification for FDA Identified Issues
  5. The FDA Will Have Questions, Lots of Them
  6. Designate a Spokesperson to Lead the Discussion
  7. Unity is a Virtue
  8. Take Time to Summarize
  9. Call in Early
  10. Call a “Time Out” if Needed
  11. Identify yourself
  12. You May Get the FDA’s Comments Early
  13. Overall, Remain Calm, Be Factual and Enjoy the Experience.

Read on, for my thoughts on how each of these tips can be used to ensure a smooth meeting with the FDA.

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What the Heck is a Regenerative Medicine Therapy?

My favorite working definition for regenerative medicine is any medical intervention that is intended to repair, replace, restore or regenerate (the 4 R’s) the cellular, tissue or organ systems in the human4Rs.jpg body. As described by one of my former FDA colleagues, Dr. Steven Bauer, cellular therapies represent one branch of regenerative medicine that on their own can fulfill the 4 R’s. However, it is clear that many other medical technologies will contribute to the field of regenerative medicine. See my post on how the FDA views the world of regenerative medicine.

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How Does the FDA View the World of Regenerative Medicine?

A major focus of the Regenerative Medicine Guru Blog is the development of new medical interventions based on regenerative medicine.

Therefore, it is important to understand how the ‘gatekeepers’ view this field. In the U.S., the Food and Drug Administration (FDA) is the key gatekeeper. You may be surprised to learn that there is not a specific regulatory pathway spelled out for regenerative medicine products.

Instead, the FDA’s approach is to examine each specific regenerative medicine product and determine the regulatory pathway based on the most important component(s) of the regenerative medicine product. To accomplish this, the FDA essentially conducts a thought experiment in which the regenerative medicine product is ‘de-constructed’ in order to identify the key components and ask the question, which component(s) are most important in providing benefit to the patient.

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FDA Regulation of Regenerative Medicine Products

Welcome to the Regenerative Medicine Guru Blog

The focus of this blog will be to provide information on how regenerative medicine products are regulated in the U.S.

It is intended to be a knowledge portal for those interested in the development of regenerative medicine.

We will go in depth into the process to successfully develop a regenerative medicine product in the U.S. including common pitfalls to avoid.

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