FDA Accepts Krystal Biotech’s Biologics License Application for Dystrophic Epidermolysis Bullosa
August 18 2022 - 4:00PM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in
redosable gene therapy, today announced that the US Food and Drug
Administration (FDA) has accepted for filing the Company’s
Biologics License Application (BLA) for B-VEC for the treatment of
patients with dystrophic epidermolysis bullosa (DEB). The
application has been granted Priority Review designation, and the
Prescription Drug User Fee Act (PDUFA) action date is February 17,
2023. The FDA stated that it is not currently planning to hold an
advisory committee meeting to discuss the application.
"We are delighted to receive the FDA’s acceptance of our
BLA submission and move one step closer to potentially bringing a
medicine to fundamentally treat DEB patients,” said Suma
Krishnan, Co-Founder and President, Research & Development of
Krystal Biotech. “We are committed to working closely with the FDA
to bring this potential, first-ever treatment to patients living
with DEB as quickly as possible.”
The BLA submission for B-VEC is supported by data from two
placebo controlled clinical trials - the GEM-3 trial (NCT04491604)
and the GEM-1/2 trial (NCT03536143).
Priority Review designation is granted to applications for
medicines that treat a serious condition, and if approved, would
provide a significant improvement in the safety or effectiveness of
the treatment, prevention, or diagnosis of a serious condition.
B-VEC was previously granted Fast Track designation and
Regenerative Medicine Advanced Therapy (RMAT) by the FDA for the
treatment of DEB.
The Company plans to submit a marketing authorization
application to the European Medicines Agency (EMA) in the second
half of 2022.
About Dystrophic Epidermolysis Bullosa (DEB)DEB
is a rare and severe disease that affects the skin and mucosal
tissues. It is caused by one or more mutations in a gene
called COL7A1, which is responsible for the production of the
protein type VII collagen (COL7) that forms anchoring fibrils that
bind the dermis (inner layer of the skin) to the epidermis (outer
layer of the skin). The lack of functional anchoring fibrils in DEB
patients leads to extremely fragile skin that blisters and tears
from minor friction or trauma. DEB patients suffer from open
wounds, which leads to skin infections, fibrosis which can cause
fusion of fingers and toes, and ultimately an increased risk of
developing an aggressive form of squamous cell carcinoma which, in
severe cases, can be fatal.
About B-VECB-VEC is an investigational
non-invasive, topical, redosable gene therapy designed to deliver
two copies of the COL7A1 gene when applied directly to DEB wounds.
B-VEC was designed to treat DEB at the molecular level by providing
the patient’s skin cells the template to make normal COL7 protein,
thereby addressing the fundamental disease-causing mechanism.
The FDA and EMA have each granted B-VEC orphan drug designation
for the treatment of DEB, and the FDA has granted B-VEC fast track
designation and rare pediatric designation for the treatment of
DEB. In addition, the FDA granted Regenerative Medicine Advanced
Therapy (RMAT) to B-VEC for the treatment of DEB and the EMA
granted PRIority MEdicines (PRIME) eligibility for B-VEC to treat
DEB.
About Krystal Biotech, Inc.Krystal Biotech,
Inc. (NASDAQ: KRYS) is a pivotal-stage gene therapy company
leveraging its proprietary, redosable gene therapy platform and
in-house manufacturing capabilities to develop life-changing
medicines for patients with serious diseases, including rare
diseases in skin, lung, and other areas. For more information
please visit http://www.krystalbio.com, and follow @KrystalBiotech
on LinkedIn and Twitter.
Forward-Looking StatementsAny statements in
this press release about future expectations, plans and prospects
for the Company, including statements about the timing of the
submission of the Company’s EMA marketing authorization
application, and other statements containing the words
“anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,”
“plan,” “predict,” “project,” “target,” “potential,” “likely,”
“will,” “would,” “could,” “should,” “continue,” and similar
expressions, constitute forward-looking statements within the
meaning of The Private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including: uncertainties associated with regulatory review
of clinical trials and applications for marketing approvals, the
availability or commercial potential of product candidates
including B-VEC, the sufficiency of cash resources and need for
additional financing and such other important factors as are set
forth under the caption “Risk Factors” in the Company’s annual and
quarterly reports on file with the U.S. Securities and Exchange
Commission. In addition, the forward-looking statements included in
this press release represent the Company’s views as of the date of
this release. The Company anticipates that subsequent events and
developments will cause its views to change. However, while the
Company may elect to update these forward-looking statements at
some point in the future, it specifically disclaims any obligation
to do so. These forward-looking statements should not be relied
upon as representing the Company’s views as of any date subsequent
to the date of this press release.
CONTACT:Investors and
Media:Meg DodgeKrystal Biotechmdodge@krystalbio.comSource:
Krystal Biotech, Inc.
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