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• Recessive Dystrophic Epidermolysis Bullosa (RDEB)
• Localized Scleroderma
• Arthritis and Related Conditions

Expanded Access Policy for Fibrocell Investigational Therapies

Fibrocell is a gene therapy company focused on developing autologous cell-based therapies for rare diseases affecting the skin and connective tissue. Currently, Fibrocell has two investigational gene therapies in development: FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), and FCX-013 for the treatment of moderate to severe localized scleroderma. In order to advance investigational therapies, such as FCX-007 and FCX-013, clinical trials are conducted to assess a product candidate’s safety and efficacy. After this data is collected and evaluated, a company plans to submit an application to the U.S. Food and Drug Administration (FDA) and other relevant regulatory agencies to obtain approval to commercially market the product. A company cannot provide patients with broad access to products until the FDA and other regulatory agencies grant approval.  

What is expanded access?

Presently, the only way for patients to obtain access to investigational therapies in development by Fibrocell is to participate in clinical trials. Some patients may not meet eligibility requirements for participation in these trials and consequently, may seek expanded access, which is also known as pre-approval access or compassionate use.

Expanded access enables manufacturers of drugs and biologics to obtain permission from the FDA to offer investigational products—those not yet approved by the FDA—to patients with serious or life-threatening conditions outside of a traditional clinical trial setting.

Clinical trials are conducted to understand each investigational product’s safety and efficacy profile, and appropriate dosing regimen. Fibrocell believes that participation in clinical trials is the best way for patients to obtain access to the Company’s investigational gene therapies before they are approved by the FDA and other regulatory agencies.

There may be occasions when it is not possible for patients to participate in a clinical trial. In these cases, and where patients have serious or life-threatening conditions with no satisfactory alternative treatment options, expanded access to Fibrocell’s investigational new gene therapies may be requested. The expanded access request must be submitted by a healthcare professional who is responsible for overseeing the patient’s treatment.

More information about clinical trials, including those for Fibrocell’s investigational gene therapies, may be found at https://clinicaltrials.gov. Search the word “Fibrocell” to find the Company’s clinical trials, including the status of patient recruitment.

How is expanded access requested?

• Procedure: All requests for expanded access to Fibrocell’s investigational gene therapies must be prepared and submitted electronically by the patient’s healthcare provider (i.e., physician) to the Fibrocell Patient Affairs Team at ea@fibrocell.com. The physician must be licensed in the country where the patient resides.
• Contact Information: If you are a healthcare professional and would like to request expanded access to a Fibrocell investigational gene therapy, please send an email to the Fibrocell Patient Affairs Team at ea@fibrocell.com. The following information must be included in your request: 
  • Name of investigational gene therapy being requested
  • Explanation of the reason that the investigational gene therapy is needed
  • Your contact information, including address, email address, telephone, and medical license number
  • Do not include any personally-identifiable patient information in your request
• Timeline: Fibrocell anticipates acknowledging receipt of requests for expanded access within 5 business days. Fibrocell will consider all requests for expanded access in a fair and timely manner.

How is an expanded access request evaluated?

Fibrocell will consider requests for expanded access on a case-by-case basis. Moreover, the Company relies on guidelines supplied by the FDA and other regulatory agencies, as well as other important criteria when assessing our ability to provide expanded access for our investigational gene therapies. The following criteria will be considered, including, but not necessarily limited to:

• Whether the patient is ineligible or unable to participate in an ongoing clinical trial;
• Whether the patient has a serious or life-threatening disease or condition;
• Whether there are alternative therapies available that would be satisfactory;
• Whether the investigational gene therapy is currently being studied in humans;
• Whether the patient suffers from a disease or condition that is sufficiently similar to the disease or condition for which a clinical trial is being conducted;
• Whether providing the investigational gene therapy on an expanded access basis will interfere with the conduct of clinical trials or any regulatory submissions;
• Whether there are sufficient data to show that the potential benefits of the gene therapy outweigh the possible risks, based on available safety and efficacy data;
• Whether Fibrocell plans to pursue regulatory approval for the gene therapy in the patient’s country; and/or
• Whether there is an adequate supply of the investigational gene therapy to complete ongoing and planned clinical trials.

Because the circumstances of each request are different, there is no guarantee that expanded access will be granted for any of our investigational gene therapies.

Do you have any questions?

You will find information about Fibrocell’s investigational gene therapies at www.fibrocell.com under the tab, “Pipeline & Clinical Trials.” If you have questions, please contact the Fibrocell Patient Affairs Team at ea@fibrocell.com.

Please note: This policy is subject to change at the discretion of Fibrocell and subsequently, this web page will be updated to reflect any revisions to the policy.