Krystal Biotech, Inc. (the Company) (NASDAQ: KRYS), a biotechnology
company focused on developing and commercializing genetic medicines
for patients with rare diseases, today announced the US Food and
Drug Administration (FDA) has approved VYJUVEK™ (beremagene
geperpavec-svdt) for the treatment of patients six months of age or
older with dystrophic epidermolysis bullosa (DEB). VYJUVEK is
designed to address the genetic root cause of DEB by delivering
functional copies of the human COL7A1 gene to provide wound healing
and sustained functional COL7 protein expression with redosing.
VYJUVEK is the first-ever redosable gene therapy and the first and
only medicine approved by the FDA for the treatment of DEB, both
recessive and dominant, that can be administered by a healthcare
professional in either a healthcare professional setting or in the
home.
DEB is a rare and serious disease that affects the skin and
mucosal tissues caused by one or more mutations in the COL7A1
gene. The COL7A1 gene is responsible for the production of
functional COL7 protein that forms anchoring fibrils necessary to
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
with minor friction or trauma. DEB patients suffer from open
wounds, which lead to recurrent skin infections and fibrosis that
can cause fusion of fingers and toes, and ultimately increase the
risk of developing an aggressive form of skin cancer.
“This is a devastating disease,” said M. Peter Marinkovich,
M.D., primary investigator of the GEM-3 trial, Director of the
Blistering Disease Clinic at Stanford Health Care and Associate
Professor of Dermatology at the Stanford University School of
Medicine. “Until now, doctors and nurses had no way to stop
blisters and wounds from developing on dystrophic EB patient skin
and all we could do was to give them bandages and helplessly watch
as new blisters formed. VYJUVEK topical gene therapy changes all of
this. VYJUVEK both heals patient wounds and prevents skin from
re-blistering because it actually corrects the underlying skin
defect of dystrophic EB. Because it’s safe and easy to apply
directly to wounds, it doesn't require a lot of supporting
technology or specialized expertise, making VYJUVEK highly
accessible even to patients who live far away from specialized
centers."
The FDA approval of VYJUVEK is based on two clinical studies.
The GEM-1/2 trial was an intra-patient, open label, single center,
randomized, placebo-controlled study showing that repeat topical
applications of VYJUVEK were associated with durable wound closure,
full-length cutaneous COL7 expression, and anchoring fibril
assembly with minimal reported adverse events. The GEM-3 trial
was an intra-patient, double-blinded, multi-center, randomized,
placebo-controlled study that met both its primary endpoint of
complete wound healing at six months and its key secondary endpoint
of complete wound healing at three months. VYJUVEK was well
tolerated with no drug-related serious adverse events or
discontinuations due to treatment-related events.
“Data from our GEM-1/2 trial and our GEM-3 trial, published in
Nature Medicine and the New England Journal of Medicine,
respectively, demonstrated the strength of both studies showing
that VYJUVEK safely and effectively improved wound healing,” said
Suma Krishnan, President, Research & Development, Krystal
Biotech, Inc. “For so many years, all we have been able to offer
DEB patients was palliative care, but now, based on the strength of
the Company’s clinical trial data, there is a safe and effective
FDA approved treatment.”
“Today’s landmark approval of VYJUVEK as the first redosable
gene therapy ushers in a whole new paradigm to treat genetic
diseases and is an important milestone for patients affected by DEB
as well as their families and caregivers," said Krish S. Krishnan,
Chairman and Chief Executive Officer of Krystal Biotech, Inc. "We
offer our sincere gratitude to DEB patients, caregivers,
investigators, US regulators, and our employees who made this
approval possible. For Krystal, this is a transformative
achievement that highlights our commitment to developing and
commercializing novel therapies for patients with rare diseases and
demonstrates Krystal’s capability as a fully-integrated company
ready to launch and bring VYJUVEK to patients as quickly as
possible and deliver additional transformative medicines to
patients as we advance our pipeline.”
VYJUVEK is expected to be available in the United States in the
third quarter of 2023, and the Company will begin the promotion of
VYJUVEK immediately. To meet the needs of patients, caregivers, and
families as they start and continue their VYJUVEK treatment
journey, the Company has developed Krystal Connect, a
personalized support program. The program includes resources that
can answer questions about VYJUVEK, verify health benefits, support
treatment planning and administration and provide information about
financial assistance for eligible patients. For more information,
patients, caregivers and healthcare professionals can call Krystal
Connect at 1-844-5-KRYSTAL.
With this approval, the FDA issued the Company a Rare Pediatric
Disease Priority Review Voucher (PRV), which confers priority
review to a subsequent drug application that would not otherwise
qualify for priority review. The PRV program is designed to
encourage the development of new drugs for the prevention or
treatment of rare diseases.
“With the FDA approval of VYJUVEK, the DEB population has
reached a monumental milestone in the treatment of this horrible
disorder. Our hopes have now been realized for a safe and effective
treatment for one of the most devastating symptoms of the
disorder,” said Brett Kopelan, Executive Director of debra of
America, the national organization dedicated to improving the lives
of all people living with EB in the US. Mr. Kopelan added, “we
thank Krystal for their dedication and commitment to bringing
VYJUVEK to fruition. People living with DEB will now have a
significant chance of having an improved quality of life and debra
will continue to work closely with Krystal to assure patients have
ready access to VYJUVEK.”
Outside of the US, the European Medicines Agency has granted
VYJUVEK orphan drug designation and PRIME (PRIority MEdicines)
eligibility for the treatment of DEB. The Company anticipates
starting the official Marketing Authorization Application procedure
in the second half of 2023 with a potential approval in 2024. The
Company is also working with the Pharmaceuticals and Medical
Devices Agency in Japan to study VYJUVEK and seek approval for
potential launch in 2025.
Visit VYJUVEK.com for more information, including full
Prescribing Information.
Investor Conference Call and Webcast
The Company will host an investor conference call and webcast to
discuss the FDA’s approval of VYJUVEK on Friday, May 19, 2023, at 6
pm ET.
To register and participate in the conference call, please go
to: https://www.netroadshow.com/events/login?show=d32d1c5d&confId=51401.
For those unable to listen to the live conference call, a replay
will be available on the Investors section of the Company’s website
at www.krystalbio.com.
About VYJUVEK
VYJUVEK 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 Reactions
The 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.
Contraindications
None.
Warnings and Precautions
VYJUVEK 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 US Prescribing Information.
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 regarding the timing of the
commercial availability of VYJUVEK in the U.S., Europe, and Japan
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 applications for marketing approvals,
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
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/81a6404d-d9fb-4a1c-9d03-f8fb958c4cd8
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