Wednesday, August 10, 2022

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

Kevin C. Kemp, Anastasia Georgievskaya, Kelly Hares, Juliana Redondo, Steven Bailey, Claire M. Rice, Neil J. Scolding, Chris Metcalfe & Alastair Wilkins; Nat Commun 13, 4655 (2022). doi:10.1038/s41467-022-31450-w 

 Pharmacological interventions to increase frataxin expression and reverse the deleterious effects of frataxin deficiency are attractive therapeutic approaches in FA. We have shown that a course of G-CSF therapy in participants with FA is safe, is associated with effective HSC mobilisation, and leads to significant elevations in frataxin together with improvements in biochemical deficits associated with FA. Nevertheless, the need for future assessment of G-CSF administration on affected tissues, such as the heart and brain, using a range of dose levels and dosing frequencies is required. The long-term safety of sustained G-CSF administration in people with FA is also unknown. The natural rate of disease progression in FA necessitates prolonged trial periods to sufficiently detect changes in clinical measures. This study provides proof-of-principle evidence to support an efficacy study of G-CSF administration in FA, using repeated courses over a longer period.

 

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

FDA Extends Review of Omaveloxolone in Friedreich Ataxia

August 9, 2022. After Reata Pharmaceuticals submitted an analysis from the MOXIe extension study of the investigational agent as part of the NDA submission, the FDA extended the PDUFA date to February 2023.