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Diroximel fumarate (DRF)(ALKS8700, BIIB098; Alkermes, Waltham, MA and Biogen, Cambridge, MA) is an oral fumarate in development for treatment of multiple sclerosis (MS). Like dimethyl fumarate (DMF) (Tecfidera; Biogen, Cambridge, MA), DRF is a prodrug that is converted to monomethyl fumarate. The unique chemical structure of DRF, however, creates potential for similar efficacy with better gastrointestinal tolerability; this is supported by interim data from week 48 of a 96-week open-label safety study (NCT02634307) presented at the American Academy of Neurology May 4-10 in Philadelphia,...
A prodrug formulation of levodopa-carbidopa delivered as an intestinal gel (LCIG) (Duopa; Abbvie, Chicago, IL) is approved by the Food and Drug Administration (FDA) for treatment of motor fluctuations and dyskinesia in advanced Parkinson’s disease (PD). Interim analyses of data from a multisite international prospective study (NCT02611713) confirm that treatment with LCIG decreases “OFF” time (mean decrease -4.1 hours from baseline over 12 months). Dyskinesia was also reduced, as measured by a -12.7-point decrease from baseline on Unified Dyskinesia Rating Scale...
Results from multiple trials of SMN1gene-replacement therapy (Zolgensma; Avexis, Bannockburn, IL) show promise for single-dose treatment of spinal muscular atrophy (SMA). In the STRIVE phase 3 study for treatment of spinal muscular atrophy, 22 infants under age 6 months, diagnosed genetically (biallelic SMN1 mutations/deletions, 2 SMN2 copies), were treated. Of the 15 babies treated more than 13.6 months or who discontinued the study before then, 13 (87%) survived without needing permanent ventilation, in contrast to the 25-50% survival rate for children with SMA when untreated....
Bob Azelby, MBA
Deena Kuruvilla, MD
Katharine Nicholson, MD