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Krystal Biotech (NASDAQ:KRYS) said on Monday that the U.S. Food and Drug Administration (FDA) extended the review period by three months of its application seeking approval for its gene therapy B-VEC to treat patients with a type of skin disorder called dystrophic epidermolysis bullosa (DEB).
Krystal added that on Jan. 5, the FDA notified the company that based on manufacturing information submitted to the agency on Dec. 20, 2022, in response to an information request, the decision was taken.
The FDA considered the new information submitted as a major amendment to the biologics license application (BLA) which will require additional time for review, the company noted.
Krystal said that 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, according to the company.
The FDA is now expected to make a decision by May 19, compared to the prior action date, which was by Feb. 17. The FDA had granted priority review to the BLA.
Krystal added that proposed labeling discussions will be held no later than April 20.
The company noted that the BLA late-cycle review meeting was completed on Dec. 15, 2022 and the FDA had then indicated that there will be no Advisory Committee meeting for B-VEC and a Risk Evaluation and Mitigation Strategies program is not needed for the B-VEC application.
All pre-approval inspections of clinical sites and internal manufacturing and testing facilities have been successfully completed, according to the company.
"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 Krystal Chairman and CEO Krish Krishnan.
DEB is a rare disease in which the skin becomes fragile and can tear or blister from minor injury or friction and is caused by one or more mutations in a gene called COL7A1.