Krystal
Biotech's beremagene geperpavec (B-VEC) has been authorized by the FDA for
treating wounds of dystrophic epidermolysis bullosa (DEB).
United
States: On Friday,
19th May 2023, Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene
therapy made by Krystal Biotech, Inc., has been approved by the US FDA for the
treatment of wounds in patients who are 6 months or older and are suffering with
dystrophic epidermolysis bullosa (DEB) and mutations in the collagen type VII
alpha 1 chain (COL7A1) gene. DEB is a hereditary condition that damages
connective tissue in the skin and nails and is caused by mutations in the
COL7A1 gene. This gene codes for type VII collagen (COL7), an important protein
that serves to build and stabilize the skin's outer and middle layers. When
COL7A1 levels are low, skin layers might split, resulting in painful blisters
and ulcers.
Vyjuvek has been genetically
engineered to deliver normal copies of the COL7A1 gene to the wounds and has
also been altered such that it can no longer multiply in normal cells. Prior to
topical use, Vyjuvek is blended into an excipient (non-active component) gel
and is applied once a week by a healthcare worker as droplets to patient's
wounds. Vyjuvek's safety and efficacy
were demonstrated principally in a randomized, double-blind, placebo-controlled
research comprising 31 people with DEB, including 30 with recessive dystrophic
epidermolysis bullosa (RDEB) and one with dominant dystrophic epidermolysis
bullosa (DDEB).
Krystal Biotech's biologics
licence application (BLA) for B-VEC (Vyjuvek) was supported by results from two
intrapatient, placebo-controlled clinical trials: the phase 2 GEM-1/2
(NCT03536143) study and the phase 3 GEM-3 (NCT04491604) research.
With the approval of the
topical gene therapy, Vyjuvek will be accessible in the United States from the
third quarter of 2023, and the Company will begin marketing it immediately.
Krystal Connect, a personalized support programme, is also designed by the
Company to fulfil the requirements of patients, caregivers, and families as
they begin and continue their Vyjuvek treatment journey. The programme
comprises services that may answer Vyjuvek-related queries, verify health
benefits, aid with treatment planning and administration, and give information
about financial assistance for qualified patients.
According to the Founder
& CEO of Krystal Biotech, Inc., "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,". "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."
According to TechSci Research,
with a broad
range of therapies, Krystal Biotech Inc., is a major player in the field of
gene therapy. B-VEC, the company's flagship product, is a ground-breaking
treatment for Dystrophic Epidermolysis Bullosa. With its singular emphasis on
creating genetic treatments for rare diseases, Krystal Biotech is strategically
positioned in the biotech industry. The potential of Krystal Biotech's advanced
pipeline and a strong foundation based on scientific rigor and strategic
execution is driving the company's financial outlook in a positive direction.
The uniqueness of B-VEC's strategy and the significant unmet need in DEB
treatment present a fascinating opportunity to treat a rare hereditary disorder
and is expected to drive market expansion after the FDA approval.
The approval of
Krystal's treatment B-VEC marks a series of "firsts" in gene therapy
and exemplifies the increasing focus on gene therapy techniques to treat
serious diseases with significant unmet medical need. Doctors and nurses have been unable to
prevent blisters and wounds from forming on the skin of dystrophic EB patients
up to this point, but topical gene therapy with VYJUVEK may come up as the game
changer in the treatment of DEB. This is the first time that a topical,
non-invasive gene therapy which is redosable is approved by FDA. It would also
be the first time for any company to develop a gene therapy for a rare genetic
disease using a viral vector (Herpes Simplex Virus Type 1 — HSV1) other than
[adeno-associated virus] or lentivirus was approved. It is the sixth gene
treatment for a rare genetic condition that the FDA has approved so far, as up
to five more gene therapies for rare genetic diseases could receive FDA
approval in 2023. Thus, more gene therapy product is expected to expand its
reach to a wider range of diseases, including complex genetic disorders,
infectious diseases, cancer, neurodegenerative disorders, and cardiovascular
diseases as topical form of drugs are convenient and redosable, the demand for
the topical drugs is likely to rise. Moreover, advances in delivery systems,
gene editing technologies like CRISPR-Cas9, and a deeper understanding of
disease genetics will contribute to expanding the applicability of gene therapy
in the upcoming years.