Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
05 September 2023 - 1:00PM
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, announced today that the
U.S. Food and Drug Administration (FDA) granted Orphan Drug
Designation for KB408 for the treatment of alpha-1 antitrypsin
deficiency (AATD).
AATD is caused by mutations in the SERPINA1 gene that
lead to decreased levels and/or decreased functionality of alpha-1
antitrypsin protein. The protein is primarily produced in the liver
and secreted into the bloodstream, where it acts as a circulating
serine protease inhibitor whose principal substrate is neutrophil
elastase in the lungs. Over time, the deficiency can lead to
progressive enzymatic destruction of the lung tissue, ultimately
causing life-threatening pulmonary impairment and severe
respiratory insufficiency. In severe cases, current disease
management includes intravenous augmentation therapy which requires
weekly infusions, the clinical benefit of which remains to be
established.
KB408 is an inhaled (nebulized) formulation of the Company’s
novel replication-defective, non-integrating HSV-1-based vector
designed to deliver two copies of the SERPINA1 transgene, that
encodes for human alpha-1 antitrypsin protein, for the treatment of
AATD.
"This important designation is a milestone in the advancement of
KB408, and this decision by the FDA underscores the need for
potential new treatment options for patients with AATD,"
said Suma Krishnan, President, Research & Development,
Krystal Biotech, Inc. "We are encouraged by the profile of KB408 in
preclinical studies to date and look forward to dosing patients
once we receive clearance from the FDA."
About Orphan Drug DesignationOrphan Drug
Designation is granted by the FDA to investigational therapies
addressing rare medical diseases or conditions that affect fewer
than 200,000 people in the U.S. Orphan drug status provides
benefits to drug developers, including assistance in the drug
development process, tax credits for clinical costs, exemptions
from certain FDA fees and seven years of post-approval marketing
exclusivity.
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 the expected
timing of dosing patients with KB408, 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 Dodge
Krystal Biotech
mdodge@krystalbio.com
Krystal Biotech (NASDAQ:KRYS)
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