Krystal Biotech Receives European Commission Orphan Designation for KB407 for the Treatment of Cystic Fibrosis
January 18 2023 - 7:00AM
Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a
biotechnology company focused on developing and commercializing
genetic medicines for patients with rare diseases, announced today
that the European Commission has granted orphan designation for
KB407 for the treatment of Cystic Fibrosis (CF).
KB407 is an investigational, redosable gene therapy designed to
correct the underlying cause of cystic fibrosis by delivering two
copies of the CFTR gene directly to the airway epithelial cells
when delivered via a nebulizer. By inducing expression of full
length, normal CFTR protein in the lung, treatment with KB407 has
potential to restore ion and water flow into and out of lung cells
to correct the lung manifestations of the disease in patients
regardless of their underlying genetic mutation.
“We are pleased that the European Commission has granted orphan
drug designation to KB407, which we believe is an important step
toward advancing this option to treat patients with CF,” said Suma
Krishnan, President of Research and Development at Krystal
Biotech.
The U.S. Food and Drug Administration has also granted Orphan
Drug designation for KB407 to treat patients with CF.
About European Commission Orphan
Designation
Orphan drug designation in the European Union (EU) is granted by
the European Commission based on a positive opinion issued by the
European Medical Association (EMA) Committee for Orphan Medicinal
Products. The EMA’s orphan designation is available to companies
developing treatments for life-threatening or chronically
debilitating conditions that affect fewer than five in 10,000
persons in the EU. Medicines that meet the EMA’s orphan designation
criteria qualify for financial and regulatory incentives that
include a 10-year period of marketing exclusivity in the EU after
product approval, protocol assistance from the EMA at reduced fees
during the product development phase and access to centralized
marketing authorization.
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
orphan drug designation for KB407 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 KB407, 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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