Drugs Awaiting FDA Approval for Multiple Sclerosis
Currently, there are no specific drugs awaiting FDA approval for multiple sclerosis that are documented in the available evidence, though several newer-generation sphingosine-1-phosphate (S1P) receptor modulators are under investigation and expected to enter the treatment arena soon. 1
Current FDA-Approved Landscape
The FDA has recently expanded the MS treatment arsenal with several approvals:
Ublituximab was FDA-approved as an intravenous glycoengineered chimeric anti-CD20 IgG1 monoclonal antibody for relapsing forms of MS, based on the ULTIMATE I and II phase 3 trials showing superiority versus teriflunomide 2
Ocrelizumab received FDA approval for both relapsing MS and primary progressive MS, as the OPERA 1 and 2 trials demonstrated reduced brain atrophy compared to subcutaneous interferon beta-1a 3
Cladribine was approved by the European Medicines Agency (EMA) in 2017 after submitting new efficacy and safety data, and in August 2018, the FDA agreed to re-evaluate cladribine data as a treatment option for relapsing-remitting MS after its initial rejection seven years earlier due to safety concerns 1
Drugs Under Investigation
The evidence indicates ongoing research rather than imminent approvals:
Newer-generation S1P receptor modulators are currently being investigated and are expected to become available soon, though specific agents and timelines are not detailed 1
Anti-LINGO-1 monoclonal antibody is being explored as a neuroprotective treatment for progressive MS, representing a novel approach targeting remyelination rather than inflammation alone 4
Important Context on Treatment Gaps
The paramount challenge in MS remains bridging the disconnect between anti-inflammatory therapies and ineffective neuroprotective strategies, necessitating a dual-target approach. 5 Current disease-modifying therapies—including interferon-β, glatiramer acetate, oral S1P modulators, fumarates, teriflunomide, cladribine, natalizumab, and anti-CD20 monoclonals (ocrelizumab, ofatumumab, ublituximab)—reduce relapse rates and MRI activity but do not consistently prevent disability progression, particularly in non-active progressive MS 5
Clinical Implications
For aggressive relapsing-remitting MS refractory to high-efficacy disease-modifying therapies, Autologous Hematopoietic Stem Cell Transplantation (AHSCT) using intermediate-intensity conditioning achieves superior disease control compared to continued DMT escalation 6
The focus of current research is identifying molecules with high efficacy for progressive forms of MS, as future efforts must concentrate on understanding pathogenetic mechanisms underlying disease progression to characterize novel therapeutic targets 4