Mark Walters, MD, on Issues Facing First Possible Sickle Cell Gene Therapy Approval


The professor in residence of pediatrics at University of California San Francisco discussed factors that will limit the potential first approval, including access and policy.

“[Gene therapy] cannot have an impact on sickle cell disease unless persons who have severe disease, and are in pain much of the time, can get access to it. And if they can't get access to it, its impact will be limited. Many in the community are anxious about this, because the price point will be challenging, I predict. And most of my patients, and I think this is true across the country, have public insurance."

Lovotibeglogene autotemcel (lovo-cel; bluebird bio) gene therapy is up for FDA review for the treatment of sickle cell disease (SCD) with a Prescription Drug User Fee Act (PDUFA) date of December 20, 2023.1 While the possible approval has the potential to transform the treatment landscape of SCD, such a change comes with its own challenges and questions, and more research will be ongoing to address these.

CGTLive spoke with Mark Walters, MD, professor in residence, pediatrics, Sickle Cell Center of Excellence, University of California San Francisco, an investigator on the phase 1/2 HGB-205 (NCT02151526) and HGB-206 (NCT02140554) clinical trials evaluating lovo-cel, to learn more about his thoughts on lovo-cel and its possible approval. While he believes that the therapy will be approved, he stressed that a lot of work must be done to improve the accessibility of gene therapy to a wider population. He touched on health care policy and insurance as 2 important pieces of the access puzzle.


bluebird bio confirms that FDA has communicated that advisory committee meeting will not be scheduled for lovo-cel gene therapy for sickle cell disease. News release. bluebird bio. August 16, 2023.

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