Open engagement and frequent communication with regulators is needed throughout the development process for cell and gene therapies to help bring them to market, according to an October 13 session at the Cell & Gene Therapy Meeting on the Mesa.
CBER director Peter Marks praised Janet Woodcock’s work as acting FDA commissioner, and while noting that Biden needs to nominate someone to fill the role permanently by Nov. 16, he said he has “no idea” when that actually might occur. “Dr. Woodcock has been at the agency for over three decades and she, during that time, has proven herself to be a remarkably capable manager,” Marks said at the Alliance for Regenerative Medicine’s meeting Tuesday.
The US Food and Drug Administration has not been stuck in neutral while waiting for a new permanent commissioner, according to Center for Biologics Evaluation and Research Director Peter Marks. In fact, Marks said that he cannot tell the difference between operations with a permanent commissioner and Acting Commissioner Janet Woodcock.
According to a March 30 report from the Alliance for Regenerative Medicine, nearly $20 billion of investment flowed into biotech companies developing cell-, gene- and tissue-based therapies last year, more than double the figure from the previous year.
As if it were needed, Amicus Therapeutics Inc.’s spin-off of its gene therapy work and PDUFA VII’s provisions to increase the capacity of the FDA’s Center for Biologics Evaluation and Research offered further proof this week of the global explosion that’s happening in the regenerative medicine field.
The amount of academic and early-stage biotech research in this area has skyrocketed over the last few years. According to data from the Alliance for Regenerative Medicine in January this year, there are currently 1,220 ongoing clinical trials in this space, 152 of which are at phase III. Despite the global pandemic, investment in this area is also at a record high around the world, with the equivalent of €15.7B invested in 2020, a figure double that of 2019.
The House Ways & Means Committee on Wednesday (Sept. 15) passed as part of its reconciliation bill a provision that limits the number of rare disease drugs that can qualify for tax credits under the Orphan Drug Act, despite rare disease organizations’ assertions the provision would disincentivize research and could limit patients’ access to life-saving therapies.
Janet Lambert, CEO of the Alliance for Regenerative Medicine, discusses CRISPR, the surge of investment and the progress being made in developing treatments.
