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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?
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:
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.