FDA to Advance Gene Therapy Development for Certain Diseases

Significant progress has been made in gene therapy research and development over the years, with promise to treat and cure some of the most complex diseases.

In a statement, FDA Commissioner Scott Gottlieb, MD, announced the agency’s latest efforts to speed up the path to approval for gene therapies, specifically for certain specialty and rare diseases.

To help expedite advancement in the field, the FDA has released a new policy framework for the development, review, and approval of gene therapies to address the challenges of bringing these drugs to market.

According to Dr Gottlieb, the latest framework includes 6 scientific guidance documents intended to serve as the building blocks of a modern, comprehensive framework for how the agency is going to advance the field of gene therapy while making sure new products meet the agency’s standards.

In 2017, the FDA approved the first gene therapy, tisagenlecleucel (Kymriah) for refractory B-cell precursor acute lymphoblastic leukemia, followed by the approvals of axicabtagene ciloleucel (Yescarta) for diffuse large B-cell lymphoma and voretigene neparvovec-rzyl (Luxturna) for the treatment of a rare vision loss disease.

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