Fibrocell Announces FDA Fast Track Designation of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa
January 05 2017 - 7:00AM
Fibrocell Science, Inc. (NASDAQ:FCSC), a gene therapy company
focused on transformational autologous cell-based therapies for
skin and connective tissue diseases, today announced that the U. S.
Food and Drug Administration (FDA) has granted Fast Track
designation to FCX-007, the Company’s clinical-stage candidate for
the treatment of Recessive Dystrophic Epidermolysis Bullosa
(RDEB)—a rare, devastating genetic skin disease for which no
FDA-approved therapies exist.
“Fast Track designation represents a significant
milestone in advancing clinical development of FCX-007 for the
treatment of RDEB,” said John Maslowski, Chief Executive Officer of
Fibrocell. “We are pleased the Agency has awarded this designation
to FCX-007 which, we believe, has the potential to be the first
gene therapy to treat the underlying cause of RDEB and to bring
relief to patients suffering from this debilitating, painful
disease.”
The Fast Track program is designed to facilitate
development and expedite review of new therapies that address unmet
medical needs of patients with serious conditions. The
designation offers various benefits, including more frequent
meetings with the FDA to discuss the drug's development plan,
eligibility for Accelerated Approval or Priority Review if relevant
criteria are met, and opportunity for Rolling Review, which allows
the Company to submit completed sections of its Biologics License
Application (BLA) to the FDA, rather than waiting until every
section of the BLA is completed before the entire application can
be reviewed.
FCX-007 was previously granted Orphan
Designation by the FDA for the treatment of Dystrophic
Epidermolysis Bullosa (DEB), which includes RDEB, and Rare
Pediatric Disease Designation by the FDA for treatment of RDEB.
Fibrocell is developing FCX-007 in collaboration
with Intrexon Corporation (NYSE:XON), a leader in synthetic
biology.
About FCX-007
FCX-007 is Fibrocell's clinical-stage,
gene-therapy product candidate for the treatment of recessive
dystrophic epidermolysis bullosa (RDEB), a congenital and
progressive orphan skin disease caused by the deficiency of the
protein type VII collagen (COL7). FCX-007 is a
genetically-modified autologous fibroblast that encodes the gene
for COL7 and is being developed in collaboration with Intrexon
Corporation. By genetically modifying autologous fibroblasts
ex vivo to produce COL7, culturing them and then treating wounds
locally via injection, FCX-007 offers the potential to address the
underlying cause of the disease by providing high levels of COL7
directly to the affected areas while avoiding systemic
distribution.
About Recessive Dystrophic Epidermolysis Bullosa
(RDEB)
Recessive dystrophic epidermolysis bullosa
(RDEB) is the most severe form of dystrophic epidermolysis bullosa
(DEB), a congenital, progressive, devastatingly painful and
debilitating genetic disorder that often leads to death. RDEB is
caused by a mutation of the COL7A1 gene, the gene which encodes for
type VII collagen, a protein that forms anchoring fibrils.
Anchoring fibrils hold together the layers of skin, and without
them, skin layers separate causing severe blistering, open wounds
and scarring in response to friction, including normal daily
activities like rubbing or scratching. Children who inherit
the condition are often called "butterfly children" because their
skin is as fragile as a butterfly's wings. We estimate there
are approximately 1,100 – 2,500 RDEB patients in the U.S.
Currently, treatments for RDEB address only the sequelae, including
daily bandaging, hydrogel dressings, antibiotics, feeding tubes and
surgeries.
About Fibrocell
Fibrocell is an autologous cell and gene therapy
company translating personalized biologics into medical
breakthroughs for diseases affecting the skin and connective
tissue. Fibrocell’s most advanced product candidate, FCX-007,
has begun a Phase I/II trial for the treatment of recessive
dystrophic epidermolysis bullosa (RDEB). Fibrocell is in
pre-clinical development of FCX-013, its product candidate for the
treatment of linear scleroderma. In addition, Fibrocell has a
third program in the research phase for the treatment of arthritis
and related conditions. Fibrocell’s gene-therapy portfolio is
being developed in collaboration with Intrexon Corporation
(NYSE:XON), a leader in synthetic biology. For more
information, visit www.fibrocell.com or follow us on Twitter at
@Fibrocell.
Trademarks
Fibrocell, the Fibrocell logo, Fibrocell Science
and LAVIV® are trademarks of Fibrocell Science, Inc. and/or its
affiliates. All other names may be trademarks of their
respective owners.
Forward-Looking Statements
This press release contains, and our officers
and representatives may from time to time make, statements that are
“forward-looking statements” within the meaning of the safe harbor
provisions of the U.S. Private Securities Litigation Reform Act of
1995. All statements that are not historical facts are hereby
identified as forward-looking statements for this purpose and
include, among others, statements relating to: the potential
advantages of FCX-007 and Fibrocell’s other product candidates; the
potential benefits of Fast Track Designation, Orphan Designation
and Rare Pediatric Disease Designation; and other statements
regarding Fibrocell’s future operations, financial performance and
financial position, prospects, strategies, objectives and other
future events.
Forward-looking statements are based upon
management’s current expectations and assumptions and are subject
to a number of risks, uncertainties and other factors that could
cause actual results and events to differ materially and adversely
from those indicated herein including, among
others: uncertainties and delays relating to the initiation,
enrollment and completion of pre-clinical and clinical trials;
whether pre-clinical and clinical trial results will validate and
support the safety and efficacy of Fibrocell’s product candidates;
Fibrocell’s ability to obtain additional capital to continue to
fund operations; Fibrocell’s ability to maintain its collaboration
with Intrexon Corporation and the risks, uncertainties and other
factors discussed under the caption “Item 1A. Risk Factors” in
Fibrocell’s most recent Form 10-K filing and Form 10-Q filings. As
a result, you are cautioned not to place undue reliance on any
forward-looking statements. While Fibrocell may update certain
forward-looking statements from time to time, Fibrocell
specifically disclaims any obligation to do so, whether as a result
of new information, future developments or otherwise.
Investor Relations Contact
Annie Cheng
646-362-8804
acheng@clermontpartners.com
Media Relations Contact
Karen Casey
484-713-6133
kcasey@fibrocell.com
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