The development of gene therapy as a treatment for rare and life-threatening conditions is a quickly growing area of scientific exploration. As more drug developers have begun investing in this form of research, having witnessed its potential, the FDA has seen the clear need to hire more individuals who are able to skillfully review these new therapies. They currently have over 800 gene therapy applications awaiting review and they expect that they will begin to receive 200 new applications for these type of treatments annually. The prediction, is that from these applications, the FDA will be able to give approval to 10 to 20 therapies each year, starting in 2025.
In order to accomplish this rate of approval, the organization has announced their plan to hire a minimum of 50 new drug reviewers to specifically assess gene and cell therapies.
The Revolution of Gene Therapy
The FDA sees the accelerated development of gene therapies as a revolutionary advancement.
The recent growth in this type of treatment is similar to the rise in development of antibody drugs during the late 1990s. Following this rise, monoclonal antibodies became the backbone of modern treatment regimes.
It is exciting to think how we might see gene therapy advance in the next few years.
2019 Goals from the FDA
In addition to hiring new experts in gene therapy, the FDA has also released some of their other plans for this coming year.
First, they plan to “introduce new guidance” for various areas of their approval including regenerative medicine advanced therapy designation and accelerated approvals.
Accelerated approval is granted for drugs who have a predicted clinical benefit for populations of patients with a largely unmet medical need. This predicted benefit is based on a surrogate endpoint. Following accelerated approval, the drugs potential must be proved in a confirmatory trial. If this trial does not confirm its potential, it risks having its approval rescinded. However, for those living with a condition which has few or no current treatment options, accelerated approval can be life-altering.
Additionally, the FDA has specific plans for hemophilia and neurodegenerative diseases. For instance, for hemophilia, they want to encourage individual researchers to collaborate with each other, and provide them guidance to do so. By facilitating the pooling of data which followed the same manufacturing protocols, the FDA hopes to accelerate approvals for this disease.
Lastly, for CAR-T therapies, the FDA hopes to provide guidance on how new manufacturing techniques this year.
The Current State
Unfortunately, many of these larger goals have been put on hold due to the government shutdown. While we are in this state of limbo, the FDA is focusing their funds and resources on ensuring their most critical duties are performed. This means many of their desired advancements have not made progress yet this year.
The FDA plans to be very open with the public about what they’re able to complete during the shutdown and how they’re approaching the challenges at hand. They say that for now, their review of gene therapy applications is being funded by a very limited supply of carryover user fee balances.
But rest assured, big changes will be coming soon, and hopefully they will bring life-changing treatments to those living with rare and life-threatening conditions in the near future.
You can read more about the FDA’s plans for 2019 and how they may impact the research and development of new therapies for rare diseases here.