P232 Bioavailability and bioequivalence of Risdiplam tablets in healthy volunteers

Risdiplam (EVRYSDI ®) is a centrally and peripherally distributed, oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved for the treatment of spinal muscular atrophy (SMA) in over 90 countries worldwide. Currently, risdiplam is administered as an oral solution, which provides flexibility for tail oring the dosing regimen in paediatric patients. However, all patients with a body weight ≥20 kg receive a 5 mg dose. The oral solution is constituted from powder by a pharmacist and, for a dose of 5 mg, a volume of 6.6 mL has to be drawn up in an oral syringe by the patient or their caregiver eve ry day.

Source: Neuromuscular Disorders - October 1, 2023 Category: Neurology Authors: H. Kletzl, K. Heinig, B. Jaber, B. Lomeli, W. Yeung, A. Young, H. Coleman, D. Morrison Source Type: research