Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.

Although the CRL delayed the cell therapy’s approval by almost two years, additional CMC-related testing requested by the FDA ultimately came to be seen as a good thing by sponsor CellTrans because it provided assurance of product consistency.

FDA's review of documents and source data files to assess product efficacy and subject safety was complicated by missing and inconsistent data, the clinical reviewer said, concluding there was insufficient data monitoring during clinical studies or in preparing the documents for regulatory submission.

Despite a host of challenges with donislecel’s development and BLA, the US FDA approved based on clinical data from only 30 patients, using a clinically meaningful insulin independence endpoint and in a more carefully tailored population. Pink Sheet’s Drug Review Profile takes a deep dive into the FDA review of the first approved allogenic pancreas islet cell product.