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FDA Official Urges Wealthy Nations to Align Gene Therapy Rules

Nov. 4, 2019, 10:23 PM

Patients from low- and middle-income countries will have better access to gene therapies if the U.S. and other high-income countries align their regulations for these potentially curative treatments, the FDA’s biologics chief said.

“We really want to see the delivery of safe and effective gene therapies, not just in high income countries but across the globe,” Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration, said Nov. 4 at the American Society for Gene and Cell Therapy’s policy summit.

If wealthier nations eliminate inconsistencies that make it harder to bring cutting-edge therapies onto the market, it will make it easier for countries that don’t have the regulatory infrastructure in place currently for gene therapy products to make them available to their citizens, Marks said.

“Hopefully that could facilitate commercial availability and the use of gene therapy in low- and middle-income countries,” Marks said.

A handful of gene therapy products have come on the market in the past few years, such as Novartis’s Zolgensma to treat a rare, muscle-destroying disease known as spinal muscular atrophy, and Spark Therapeutics’ Luxturna to treat an inherited form of blindness.

There’s a robust pipeline of products under development, Marks said. There are more than 800 active investigational new drug applications underway, and the FDA expects to approve 10 to 20 cell and gene therapy products a year by 2025.

But some nations in Africa and Asia don’t have the regulatory infrastructure to bring these complex specialty treatments to their patients, Marks said.

The FDA is working with the World Health Organization to produce a white paper on cell and gene therapies for low- and middle-income countries. The paper won’t dictate what laws to put in place, but it will outline considerations to undertake to get familiar with cell and gene therapy products.

The World Bank defines a middle-income country as having a per capita gross national income of $1,026 to $12,475.

“Now’s the time to do it,” Marks said about putting educational processes in place for nations in Africa and some parts of Asia either about how to regulate or how to create a reference system that other regulatory systems could rely on.

“I think it’s going to be very hard five years from now when there are products that would be very relevant to get those to people in those areas,” he said.

Greatest Public Health Impact

Making gene therapy products available in lower- and middle-income countries that don’t have the supportive care that’s available in wealthier nations could have the greatest impact on public health, he said.

For example, an inherited blood disorder called beta thalassemia can be treated with aggressive supportive care, namely blood transfusions and iron chelation therapy, which reduce extra iron in the body. If a gene therapy was made available to treat the disorder, it could help patients in those countries who don’t have access to that supportive care.

“If you’re somebody in certain places in Africa, where beta thalessemia is not uncommon, most people with this disease die by age 3,” Marks said.

The FDA has been working with its counterparts in Canada and Europe toward greater regulatory consistency, Marks said, “We’ll continue to move that forward in an more active manner.”

As part of that effort, the FDA will allow regulators from other nations to sit in on early-stage development meetings with gene therapy developers and the U.S agency, Marks said.

To contact the reporter on this story: Jeannie Baumann in Washington at jbaumann@bloomberglaw.com

To contact the editors responsible for this story: Fawn Johnson at fjohnson@bloomberglaw.com; Brent Bierman at bbierman@bloomberglaw.com