Krystal Biotech Receives Orphan Drug Designation from the Japanese MHLW for Beremagene Geperpavec-svdt (B-VEC)
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Orphan Drug Designation (ODD) by the Japan Ministry of Health, Labour and Welfare for Krystal Biotech's B-VEC represents a pivotal stride in the realm of genetic medicines for dystrophic epidermolysis bullosa (DEB). This status not only accelerates the regulatory process but also avails a suite of incentives, including financial support, which is instrumental in mitigating the high costs associated with drug development in rare diseases.
Given the rarity of DEB and the scarcity of effective treatments, B-VEC's progression has the potential to fulfill a profound unmet medical need. The market response to such advancements is typically positive, as they signal both corporate momentum and potential future revenue streams. Investors should, however, remain cognizant of the inherent risks in biotech, particularly in the clinical efficacy and safety profiles that will emerge from ongoing trials.
The Japanese healthcare market is known for its stringent regulatory environment, making the ODD a significant endorsement of B-VEC's potential. Krystal Biotech's ability to navigate this process is indicative of their strategic global outlook. For stakeholders, this suggests a robust regulatory strategy that could be leveraged in other markets.
Moreover, the support from the Japanese government can reduce the timeline to market, a critical factor in competitive positioning. While this news is promising, it is essential to place it within the broader context of global drug approvals and commercialization challenges. The path from ODD to market success is fraught with regulatory hurdles and commercial execution risks, which must be expertly managed to capitalize on this opportunity.
From a financial perspective, the Orphan Drug Designation is a catalyst that can enhance investor confidence, potentially leading to a more favorable stock valuation for Krystal Biotech. The designation may also foreshadow positive cash flow implications through subsidies and tax incentives, reducing the financial burden on the company and possibly shortening the time to profitability for B-VEC.
Investors should monitor the company's burn rate and capital allocation to gauge whether these benefits translate into a sustainable competitive advantage. Additionally, the potential market exclusivity that accompanies ODD could allow for pricing power, which is a significant factor in the financial success of orphan drugs. However, the actual impact on the company's bottom line will depend on the drug's clinical success and the ability to navigate the commercial landscape.
PITTSBURGH, Dec. 19, 2023 (GLOBE NEWSWIRE) -- 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 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 >
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 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.
CONTACT
Investors and Media:
Meg Dodge
Krystal Biotech, Inc.
mdodge@krystalbio.com
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