As of today, the list of publicly announced RMAT’s has risen to 30. However, the U.S. FDA states it has received 108 total requests for RMAT designations and granted 40. [Read more…]
Orchard Therapeutics Receives RMAT for OTL-103 for Treatment of Wiskott-Aldrich Syndrome
Three-Year Registrational Data Set to be Presented by Year End 2019
Regulatory Submissions Planned in the U.S. and Europe for 2021
BOSTON and LONDON, July 29, 2019 — Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for OTL-103, Orchard’s ex vivo autologous hematopoietic stem cell (HSC)-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. [Read more…]
U.S. FDA Approved RMATs on the Rise
With recent news from Sangamo Therapeutics and Krystal Biotech, the count of announced RMAT (“Regenerative Medicine Advanced Therapy”) designations has risen to 30.
Krystal Biotech received an RMAT designation for its gene therapy KB103 on June 24, 2019. KB103 is being studied for its wound closure capacity in patients with severe generalized recessive dystrophic epidermolysis bullosa (“RDEB”). The FDA also granted a RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation. [Read more…]
FDA Grants RMAT To Sangamo Therapeutics’ Gene Therapy for Hemophilia A (SB-525)
The FDA has granted RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation.
According to Sangamo Therapeutics:
“We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit. “If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.”
FDA Approved RMAT Designations
To date, 30 RMAT designations have been publicly announced. The most recent additions were Krystal Biotech’s RMAT designation for KB103 on June 24, 2019 and Sangamo Therapeutics’ RMAT designation for SB-525 on July 5, 2019.
Krystal Biotech Receives RMAT Designation from FDA for Gene Therapy, KB103
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.”
Krystal Biotech
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.
