On June 24, 2019, Krystal Biotech (NASDAQ:KRYS) became the latest regenerative medicine company to announce that it has received an RMAT designation granted from the FDA for its gene therapy product, KB103. The FDA’s decision was based on positive results from the company’s Phase 2 placebo-controlled clinical trial of KB103 and an update on the results from its Phase 1 study. These studies are named GEM-1 and GEM-2.
The same day, the company announced it had initiated an underwritten public offering of $100.0 million of its common stock. Among other activities, Krystal Biotech intends to use these proceeds to advance KB103 into and through a Phase 3 clinical trial.
Krystsal Biotech’s RMAT Designation
RMAT stands for “Regenerative Medicine Advanced Therapy” designation. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.
The RMAT designation is well-matched to Krystal Biotech’s product development focus, because it is a gene therapy company focused on commercializing treatments for patients suffering from dermatological diseases.
KB103 is currently being studied for its wound closure capacity in patients with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”).
As stated by the company:
“KB103’s RMAT designation follows our recent announcement of the Priority Medicines, or PRIME designation received from the EMA, and we look forward to collaborating closely with the EMA and initiating a clinical trial in the EU in the upcoming months. The RMAT pathway is analogous to the Breakthrough Therapy designation designed for traditional drug candidates and medical devices and was specifically created by the U.S. Congress in 2016 to get important new cell therapy and gene therapy products to the patient earlier.
Just like the Breakthrough designation, it allows companies developing regenerative medicine therapies to interact with the FDA more frequently in the clinical testing process, and RMAT-designated products may be eligible for priority review and accelerated approval.”
Headquartered in Pittsburgh, PA, Krystal Biotech is planning a pivotal Phase 3 trial for KB103 planned to start before 2020.
To learn more about Krystal Biotech, view this company presentation by Suma Krishnan, Founder and Chief Operating Officer (COO).
Seeking a searchable, sortable Excel database of RMATdesignations you can use on-the-go? Access one here.
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