FDAnews Drug Daily Bulletin

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

June 18, 2019

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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