We have released exciting new coverage of the stem cell industry. Check it out and let me know what you think? [Read more…]
Abeona Becomes First Company to Secure Two RMATs
Abeona Therapeutics announced the U.S. FDA granted a Regenerative Medicine Advanced Therapy (RMAT) designation to ABO-102, the company’s AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.
It was the 16th RMAT designation awarded by the FDA and a landmark achievement in that it makes Abeona the first company to achieve two RMAT designations.
Headquartered in Dallas, TX, Abeona (NASDAQ: ABEO) is a clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases. Abeona’s first RMAT designation was for its EB-101 gene therapy for Epidermolysis Bullosa in January 2018. [Read more…]
What is the 21st Century Cures Act? | 21st Century Cures Act Summary
Passed December 13, 2016, the 21st Century Cures Act authorizes $6.3 billion in funding. It will support the development of innovative regenerative medicines, by providing substantial funding to the U.S. NIH and the FDA, among other groups.
21st Century Cures Act
In this article:
MiMedx’s AmnioFix® Injectable Awarded 15th RMAT Designation in the U.S.
In major news released today, MiMedx Group announced that their AmnioFix® Injectable was awarded RMAT status by the U.S. FDA. This is the third RMAT designation awarded by the FDA this year and the 15th RMAT overall since the designation was introduced by the 21st Century Cures Act. The “Regenerative Medicine Advanced Therapy” (RMAT) Designation was introduced on December 13, 2016, when Congress included several provisions in the 21st Century Cures Act, an act signed into law by the 114th United States Congress authorizing $6.3 billion in funding, the bulk of which is allocated for programs supported by the National Institutes of Health (NIH).
The 21st Century Cures Act has had a profound effect on the regenerative medicine industry, supporting expansion of this industry to 600+ companies worldwide. The act also accompanies accelerated regulatory pathways that have been introduced in several other countries worldwide, including Japan, South Korea, and the E.U.
Within the U.S., sponsors of cell and gene therapies are eligible to obtain an RMAT designation if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it could address unmet medical needs. [Read more…]
How does the U.S. FDA regulate cell therapies? (351 vs 361 Products)
In the United States, cellular therapies are regulated by the FDA’s Office of Cellular, Tissue, and Gene Therapies (OCTGT) within the FDA Center for Biologics Evaluation and Research (CBER).
According to the FDA, the Center for Biologics Evaluation and Research (CBER) regulates:
- Cellular therapy products
- Human gene therapy products
- Certain devices related to cell and gene therapy
CBER uses both the Public Health Service Act and the Federal Food Drug and Cosmetic Act as enabling statutes for oversight.
In the U.S., human tissues intended for transplantation are regulated by the FDA as “Human cells, tissues and cellular and tissue-based products” or “HCT/Ps.” Under U.S. law, any company that engages in the collection, processing, storage, screening/testing, packaging, or distribution of HCT/Ps must register with the FDA.