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Pharmaceuticals / Submissions and Approvals

Gene Therapy Candidate Upends Diabetes Care—But at What Cost?

June 18, 2019
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Researchers believe that they may be on the cusp of the first significant revolution in diabetic care in decades, with a gene therapy candidate that kept diabetes from developing in high-risk patients for an average of two years after just two weeks of IV treatment.

It’s a breakthrough for a  disease that hasn’t featured a lot of good, clinical news in years. An eight-year, multisite, Phase II clinical trial of 76 relatives of type 1 diabetics showed that doses of Provention Bio’s PRV-031 (teplizumab) kept diabetes at bay for an average of 48.4 months, compared to 24.4 months for patients in the placebo group.

Significant scientific questions remain—will the teplizumab work throughout a patient’s life? How does it affect later diabetes? And, perhaps most importantly, can it work at larger scales and larger timelines?

But there are also political and economic questions: Washington is outraged at the cost of medicines and the price of insulin is seen as exhibit A. A therapy that takes insulin off the table would change the equation while introducing new pricing issues.

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