Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a
commercial-stage biotechnology company focused on the discovery,
development and commercialization of genetic medicines to treat
diseases with high unmet medical needs, today announced that the
Japan Ministry of Health, Labour and Welfare (MHLW) has granted
Orphan Drug Designation (ODD) to beremagene geperpavec-svdt (B-VEC
or VYJUVEK) for the treatment of dystrophic epidermolysis bullosa
(DEB).
“Receiving ODD is an important step in expediting the
development of B-VEC in Japan and underscores the unmet needs of
patients with DEB,” said Suma Krishnan, President, Research &
Development, Krystal Biotech, Inc. “We look forward to working
closely with MHLW to bring this important treatment to patients in
Japan as soon as possible.”
The orphan drug designation system in Japan aims to support the
development of drugs for diseases that affect fewer than 50,000
patients in Japan, for which significant unmet medical need
exists. An investigational therapy is eligible to qualify for ODD
if there is no approved alternative treatment option or if there is
high efficacy or safety compared to existing treatment options
expected. Specific measures to support the development of orphan
drugs include subsidies for research and development expenditures,
prioritized consultation regarding clinical development, reduced
consultation fees, tax incentives, priority review of applications,
reduced application fees, and extended registration validity
period.
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 VYJUVEKVYJUVEK is a non-invasive,
topical, redosable gene therapy designed to deliver two copies of
the COL7A1 gene when applied directly to DEB wounds.
VYJUVEK 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.
Indication
VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based
gene therapy indicated for the treatment of wounds in patients six
months of age and older with dystrophic epidermolysis bullosa with
mutation(s) in the collagen type VII alpha 1 chain
(COL7A1) gene.
IMPORTANT SAFETY INFORMATION
Adverse ReactionsThe most common adverse drug
reactions (incidence >5%) were itching, chills, redness, rash,
cough, and runny nose. These are not all the possible side
effects with VYJUVEK. Call your healthcare provider for medical
advice about side effects.
To report SUSPECTED ADVERSE REACTIONS, contact Krystal
Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088
or http://www.fda.gov/medwatch.
ContraindicationsNone.
Warnings and PrecautionsVYJUVEK gel must be
applied by a healthcare provider.
After treatment, patients and caregivers should be careful not
to touch treated wounds and dressings for 24 hours.
Wash hands and wear protective gloves when changing wound
dressings. Disinfect bandages from the first dressing change with a
virucidal agent, and dispose of the disinfected bandages in a
separate sealed plastic bag in household waste. Dispose of the
subsequent used dressings in a sealed plastic bag in household
waste.
Patients should avoid touching or scratching wound sites or
wound dressings.
In the event of an accidental exposure flush with clean water
for at least 15 minutes.
For more information, see full U.S. Prescribing
Information.
About Krystal Biotech, Inc.Krystal Biotech,
Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company
focused on the discovery, development and commercialization of
genetic medicines to treat diseases with high unmet medical needs.
VYJUVEK® is the Company’s first commercial product, the first-ever
redosable gene therapy, and the only medicine approved by the FDA
for the treatment of dystrophic epidermolysis bullosa. The Company
is rapidly advancing a robust preclinical and clinical pipeline of
investigational genetic medicines in respiratory, oncology,
dermatology, ophthalmology, and aesthetics. Krystal Biotech is
headquartered in Pittsburgh, Pennsylvania. 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 Krystal Biotech, Inc., including statements about orphan
drug designation of B-VEC in Japan, the Company’s beliefs regarding
expediting the development of B-VEC in Japan, the Company’s plans
to bring B-VEC to patients in Japan as soon as possible, 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, 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 release.
CONTACTInvestors and Media:Meg
DodgeKrystal Biotech, Inc.mdodge@krystalbio.com
Krystal Biotech (NASDAQ:KRYS)
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