Krystal Biotech Receives Positive Opinion from EMA Pediatric Committee on the Pediatric Investigation Plan for B-VEC for the treatment of Dystrophic Epidermolysis Bullosa
22 September 2022 - 6:30AM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in
redosable gene therapy, announced today that the European Medicines
Agency (EMA) Pediatric Committee (PDCO) has adopted a positive
opinion on the Pediatric Investigation Plan (PIP) for beremagene
geperpavec (B-VEC) for the treatment of dystrophic epidermolysis
bullosa.
As part of the regulatory process for the registration of new
medicines in Europe, the EMA requires companies to provide a
PIP outlining their strategy for investigation of the new medicinal
product in the pediatric population. An approved PIP is a
prerequisite for filing a Marketing Authorization Application
(MAA). The positive opinion is based on the B-VEC non-clinical
safety program as well as data from the clinical studies conducted
in the US that will be included in the upcoming MAA.
“The approval of the PIP provides a clear path forward, and we
look forward to working with the EMA and PDCO to bring this
important treatment to the European market as soon as possible,”
said Suma Krishnan, President, Research & Development.
Based on this positive opinion, the Company would be eligible
for up to an additional two years of marketing exclusivity in the
EU, on top of the ten-year EU market exclusivity after market
approval in the EU.
The U.S. Food and Drug Administration (FDA) and EMA have each
granted B-VEC orphan drug designation for the treatment of DEB. The
FDA has granted B-VEC fast track designation, Regenerative Medicine
Advanced Therapy (RMAT) and rare pediatric disease designation for
the treatment of DEB. B-VEC is eligible to receive a Priority
Review Voucher (PRV) following approval of B-VEC in the US. The EMA
granted PRIority MEdicines (PRIME) eligibility for B-VEC to treat
DEB.
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.
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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