Krystal Biotech Announces FDA’s 3-Month Extension of BLA PDUFA Date and Regulatory Update for B-VEC to Treat Patients with Dystrophic Epidermolysis Bullosa
January 09 2023 - 8:00AM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) today
announced that on January 5, 2023, the U.S. Food and Drug
Administration (FDA) notified the Company that based on
manufacturing information submitted to the Agency on December 20,
2022, in response to an information request, the PDUFA date has
been revised to May 19, 2023, and proposed labeling
discussions to no later than April 20, 2023.
The manufacturing information submitted by the Company included
additional information about a replaced hardware unit in the
concentration step of the manufacturing process and comparability
data supporting the use of the unit. The unit did not affect
processing parameters or product contact materials. The FDA
considered this new information as a major amendment to the
application that will require additional time for review.
The BLA late-cycle review meeting was completed on December 15,
2022. During this meeting, the FDA indicated that there will be no
Advisory Committee meeting for B-VEC and a Risk Evaluation and
Mitigation Strategies (REMS) program is not needed for the B-VEC
application. All pre-approval inspections of clinical sites and
internal manufacturing and testing facilities have been
successfully completed.
“While we are disappointed that this change was viewed as a
major amendment, we are committed to working with the FDA as it
completes its review of the B-VEC application,” said Krish S.
Krishnan, Chairman & CEO at Krystal Biotech. “We will continue
our commercial readiness efforts and upon approval bring this
important treatment to DEB patients as soon as possible.”
The Company submitted the B-VEC BLA to the FDA in June 2022. The
FDA accepted the BLA in August 2022 and granted Priority
Review.
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-VEC
B-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 European Medical Agency (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 biotechnology company
focused on developing and commercializing genetic medicines for
patients with rare diseases. The Company’s wide-ranging pipeline is
based on its proprietary redosable HSV vector. Headquartered in
Pittsburgh, Pennsylvania, the Company is led by an experienced
management team, is fully-integrated and has core capabilities in
viral vector design, vector optimization, gene therapy
manufacturing and commercialization. For more information, please
visit http://www.krystalbio.com, and follow @KrystalBiotech on
LinkedIn and Twitter.
Forward-Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the Company, including statements about
commercial and launch readiness for B-VEC 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 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.com Source: Krystal
Biotech, Inc.
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