FDA to Ramp Up Efforts in Gene Therapy Development

- May 2nd, 2019

The number of gene therapy products in development is growing, and the FDA is advancing its efforts to move these therapies forward.

The US Food and Drug Administration (FDA) is gearing up to further its efforts in gene therapy development, the agency revealed in a press release on Wednesday (May 1).

As per the announcement, the FDA is preparing to better standardize review and safety reporting requirements for gene therapy protocols thanks to scientific expertise from the FDA’s Center for Biologics Evaluation and Research (CBER).

CBER has been growing to manage product development challenges in addition to working with developers to properly cope with difficulties as advancements in gene therapy continue to grow.


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“FDA has made it a priority to field a strong bench of highly skilled and knowledgeable experts in the cutting edge science behind gene therapy, including genome editing technologies, novel gene delivery methods and advanced manufacturing technologies,” the agency said.

CBER has created the INTERACT program to help product developers with innovative products that are very early stage. Developers can receive informal advice and help from experts, and sometimes may even be able to skip certain steps if they’re found to be unnecessary.

It’s taken a number of years, but, as the FDA notes, three products have been approved by the agency, effectively making gene therapy a “reality” in the United States.

Luxturna is a one time therapy for people with a specific inherited retina eye disorder. It was developed by Spark Therapeutics (NASDAQ:ONCE) and was the first directly administered gene therapy approved by the FDA. It treats child and adult patients with an eye disease that can lead to blindness.

The other two prodducts are cell-based gene therapies for blood cancers: Kite Pharma’s Yescarta therapy and Novartis Pharmaceuticals’ (NYSE:NVS) Kymriah.

Yescarta, which is a chimeric antigen receptor T-cell therapy, was the second gene therapy approved by FDA and the first for certain types of non-Hodgkin lymphoma. Kite Pharma, a division of Gilead Sciences (NASDAQ:GILD), received approval for the drug in October 2017.

Novartis Pharmaceuticals received the FDA nod in May 2018 for Kymriah to treat patients with large B-cell lymphoma.

While these are the only gene therapies currently approved by FDA, the agency notes that a number of gene therapy products are currently being developed to address unmet clinical needs, ranging from hemophilia to spinal muscular atrophy.

“The progress and demand in this field is also evident in the more than 800 active investigational new drug applications for gene therapies on file with FDA,” the FDA says.

Case in point, a Coherent Market Insights report, published in February of this year, estimates that the global cell and gene therapy market is set to grow at a compound annual growth rate of 21.9 percent, rising from US$6.02 billion in 2017 to US$35.4 billion in 2026.

Driving that growth, according to the report, will be an increase in cardiovascular disease, cancer and genetic disorders, while funding from the government and other agencies will help advance gene and cell therapy in clinical trials.

Don’t forget to follow us @INN_LifeScience for real-time news updates

Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.


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