Quotient Sciences  and Kadmon (a Sanofi company)  presented a poster at the European Pediatric Formulation Initiative (EuPFI) conference in September 2021, describing a program that was carried out by Quotient Sciences to develop a pediatric formulation for belumosudil, a ROCK2 selective inhibitor developed by Kadmon (a Sanofi company) for the treatment of immune disorders. The aim was to develop a new oral suspension formulation using age-appropriate excipients suitable for patients aged between 3 months and 12 years, which could be taken forward into a relative bioavailability assessment versus the reference adult tablet formulation.
Formulation design and excipient selection was based on literature reviews and regulatory guidance. Formulations with different combinations of suspending/thickening agents, preservative, sweetener, and flavors were assessed to identify the most promising systems. Lead flavor and sweetener combinations that improved palatability were identified in a taste assessment study, and the thickener/suspension system was developed and optimized. Belumosudil grades that had been jet-milled and pin-milled were assessed as part of the development studies, which confirmed that jet-milled belumosudil was preferred in achieving dose homogeneity. A lead formulation was selected following short-term stability testing. Process development studies enabled scale-up of the formulation for clinical trial manufacturing.
As a result, a belumosudil oral suspension that met the quality target product profile (QTPP) and was suitable for patients aged between 3 months and 12 years was successfully identified, enabling CTM manufacturing for a relative bioavailability assessment versus the reference adult tablet formulation.
1. Quotient Sciences: Mark Beville, Ashley Willson, Josephine Gray, Nazim Kanji
2. Kadmon (a Sanofi company): Olivier Schueller, Galit Regev
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