Safety and efficacy of MD1003 in patients with progressive multiple sclerosis

Takeaway

  • MD1003 treatment did not significantly improve disability or walking speed in patients with progressive multiple sclerosis (MS). In addition, high biotin serum concentrations can cause interference of laboratory tests; therefore, due to safety concerns and lack of efficacy, MD1003 is not recommended for treatment of progressive MS.

Why this matters

    All approved treatments for MS target the immune system; however, progressive MS may not be mediated by immunological activity only. Previous studies have shown a lack of significant effect of immunological drugs, such as natalizumab, in patients with progressive MS. MD1003 is a high-dose form of biotin which targets mitochondrial dysfunction, a hallmark of progressive MS. Therefore, it is important to understand the safety and efficacy of MD1003 in patients with this disease.