BERLIN—MD1003, a high-dose pharmaceutical grade biotin, is effective in the treatment of patients with progressive multiple sclerosis (MS), according to a report presented at ECTRIMS 2018. “This real-world study supports the growing body of evidence that MD1003 is an effective and safe treatment for progressive MS,” said lead author Jonathan Ciron, MD, of the Department of Neurology, CHU Toulouse, France, and colleagues.
In the 2016 MS-SPI study, MD1003 treatment was shown to be effective and well tolerated in patients with progressive MS. Notably, MD1003 reversed MS-related disease disability in 13% of patients with progressive MS. Based on these findings, MD1003 is currently being prescribed to patients with progressive MS in France under an expanded access program.
In the present study, Dr. Ciron and colleagues sought to determine the benefits, in terms of effectiveness and safety, of MD1003 in patients with primary progressive MS (PPMS) or secondary progressive MS (SPMS) in a clinical center.
MD1003 300 mg/day (100 mg tid) was prescribed to patients with PPMS or SPMS receiving care at a single center in France (CHU Toulouse) starting in January 2016. The following measures of effectiveness and safety were administered: Expanded Disability Status Scale (EDSS), timed 25-foot walk (T25W), nine-hole peg test (9-HPT), number of relapses, and gadolinium-enhancing lesions on T1-weighted images.
As of May 2018, a total of 220 patients received MD1003. The research team presented the results of the first 91 patients to complete one year of follow-up. At baseline, mean age was 59.5, 61.5% were female, 70.3% had SPMS, mean EDSS was 5.9, mean T25W was 50.7 seconds, 9-HPT in the dominant hand was 35.1 seconds, and the mean number of previous relapses was 5.1. After one year of treatment with MD1003, 19 (23%; n = 83 with data) patients experienced improvement in EDSS and 15 (23%; n = 66 with data) patients experienced 20% or greater improvement in T25W. Active disease, a clinically-defined relapse, or a gadolinium-enhancing T1 lesion was observed in nine (11%; n = 79 with data) patients. MD1003 was also well tolerated, the researchers noted.