Krystal Biotech Announces FDA’s 3-Month Extension of BLA PDUFA Date and Regulatory Update for B-VEC to Treat Patients with Dystrophic Epidermolysis Bullosa
The manufacturing information submitted by the Company included additional information about a replaced hardware unit in the concentration step of the manufacturing process and comparability data supporting the use of the unit. The unit did not affect processing parameters or product contact materials. The FDA considered this new information as a major amendment to the application that will require additional time for review.
The BLA late-cycle review meeting was completed on
“While we are disappointed that this change was viewed as a major amendment, we are committed to working with the FDA as it completes its review of the B-VEC application,” said
The Company submitted the B-VEC BLA to the FDA in
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.
B-VEC is an investigational non-invasive, topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC 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.
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about commercial and launch readiness for B-VEC 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 B-VEC, 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
Investors and Media
Source: Krystal Biotech, Inc.