Jeffrey Gelfand, MD reviewing Lublin F et al. Lancet 2016 Mar 12.

A randomized trial provides definitive evidence.

Most patients with multiple sclerosis (MS) present with its relapsing form, whereas about 10% to 15% exhibit progressive neurological disability with or without minimal relapse activity – known as primary-progressive MS. Although several licensed therapies can modify the course of relapsing MS, there are currently none for primary-progressive disease.

Fingolimod, an oral sphingosine 1-phosphate receptor modulator effective in relapsing MS, was studied in 970 patients with primary-progressive MS in a multicenter, randomized, double-blind, parallel-group study funded by the manufacturer. Patients between ages 25 and 65 received daily fingolimod or placebo and were monitored for at least 3 years.

The primary outcome was confirmed disability progression (CDP), defined as a composite endpoint based on a change — confirmed 3 months later — in the rater-based Expanded Disability Status Scale, 25-foot timed walk, or 9-hole peg test of upper limb function. There was no difference in outcome incidence between fingolimod and placebo (hazard ratio, 0.95; 95% confidence interval, 0.8–1.12; P=0.544). There was also no difference in brain-volume loss on magnetic resonance imaging between groups.

Citation(s):

Lublin F et al. Oral fingolimod in primary progressive multiple sclerosis (INFORMS): A phase 3, randomised, double-blind, placebo-controlled trial. Lancet 2016 Mar 12; 387:1075
 

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