CellTrans’ Donislecel: Insulin Independence Data May Offer Path To Market In Type 1 Diabetes
Proportion of subjects with freedom from exogenous insulin ‘might support the efficacy’ of the allogeneic islet cellular therapy, US FDA says after concluding that issues in two studies limit interpretability of primary analyses based on severe hypoglycemic events and HbA1c levels. An advisory committee will weigh clinical efficacy, safety and product characterization issues.
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CellTrans’ Donislecel Gets US FDA Panel Nod For Small Group Of Type 1 Diabetics
Benefits of independence from exogenous insulin outweighed the risks from long-term immunosuppression necessary with the allogeneic pancreatic islet cellular therapy, majority of panelists said, but they urged the FDA to limit the indication to a very small subpopulation of patients.
At Tofersen Panel Review, US FDA Shined A Light On Internal Differences Of Opinion
Agency’s presentations on Biogen/Ionis’ ALS drug included differing viewpoints from the statistical and clinical pharmacology teams; the transparency and emphasis on a multidisciplinary review approach may reflect lessons learned from missteps at the Aduhelm panel review in 2020.
BrainStorm’s ALS Treatment NurOwn, Filed Over Protest, Will Get US FDA Panel Review
A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.