Medicine & Research

Multiple Sclerosis Drugs in the Pipeline

By Ellen Whipple, Pharm.D.
The timeline of multiple sclerosis research dates back to the mid-1800s, at which point scientists were just beginning to understand the role of myelin in the brain. Over the years, many medications were analyzed in clinical trials and led to therapy approvals for both treatments of the underlying disease and for improving relapse rates. The FDA approved the first oral disease- modifying treatment for relapsing MS in 2010, and the first medication for use in children in 2018. There are a variety of medications in the MS pipeline today, with several different mechanisms of action and a range of clinical trial results.

In recent years, there have been a significant number of medications approved that are categorized as monoclonal antibodies. A monoclonal antibody mimics a naturally occurring antibody, and will use the individual’s own immune system to fight off the disease. For treatment of MS, monoclonal antibodies have quickly grown in popularity because of their ability to specifically target the disease with relatively high efficacy. Several monoclonal antibodies have been approved to date for treatment of MS, and these include natalizumab, alemtuzumab, and ocrelizumab.

Another medication, ofatumumab, has actually been on the market successfully for several years under the brand name Arzerra, which is FDA-approved for treatment of a specific type of cancer. Though the drug works similarly to ocrelizumab, ofatumumab would be administered by monthly subcutaneous injections rather than through infusions. Two phase III clinical trials have showed promising results, and even reported that relapse rates were decreased by 50 percent when compared to teriflunomide. Novartis is currently pursuing FDA approval for use in relapsing MS.

Ublituximab is another monoclonal antibody that is being studied for use in relapsing MS. Phase II research has already proven that the medication is safe, and now phase III trials are underway to determine its efficacy. Though similar to other existing medications, the manufacturer is hopeful that this medication will be able to provide the same benefit as ocrelizumab either at a lower dose or over a shorter infusion time.

Of a slightly different mechanism than the previously mentioned monoclonal antibodies, temelimab targets a specific protein that is believed to have a role in the development of MS. MS is caused in part by increased inflammation in the central nervous system, and the intended mechanism of action for temelimab would be to relieve this inflammation to prevent disease progression. The medication should work to help fix the damage to the myelin sheath that occurs in MS.

Though many recently approved medications are delivered by injection, there have been several developments for the treatment of MS, that are tablets or pills to be taken by mouth. Ozanimod is an oral, once-daily medication that was recently granted FDA approval for treatment of relapsing MS. Phase III clinical trials were published in 2019 and reflected significantly improved efficacy over interferon beta-1a therapy, which is typically considered first-line for relapsing MS. Results of phase III clinical trials showed that this medication slowed the progression of disability, and patients treated with this medication had fewer relapses. Though there are several other medications on the market that act in the same mechanism as ozanimod, this drug appears to have fewer side effects and a better safety profile. The most common side effects with this drug included symptoms of the common cold and headaches. The drug got released under the brand name Zeposia®.

International Pipeline

There are several medications that have shown positive preliminary trial results internationally, and are in the early stages of research in the United States. For example, Qizenday is a medication whose active ingredient is high-dose biotin, or vitamin B7. Qizenday has been studied in several French clinical trials, and the first American study is currently active, with an estimated study completion date of June 2023. If efficacious, Qizenday could be a relatively inexpensive treatment option for patients with progressive MS.

Simvastatin is a medication that has been used for the treatment of high cholesterol for many years. Recently, however, it has gained attention for its potential role as an adjunct therapy for MS. In 2010, a study conducted in Iran found that patients who were treated with interferon medication who received simvastatin adjunct therapy experienced a significantly lower number of disease attacks. A phase II British study that was begun in 2008 just recently published its complete results and showed that patients treated with simvastatin versus placebo had significantly better frontal lobe function after a two-year trial period.

Among the less successful mechanisms studied for the treatment of MS, idebenone is a medication that is a synthetic version of a common dietary supplement, Coenzyme Q10. Results of a three-year study conducted by the National Institutes of Health were presented in 2018 and showed no significant benefit. Another medication, ibudilast, under-went phase II clinical trials. Though the medication was found to slow the progression of brain atrophy, it was associated with much higher rates of gastrointestinal side effects.


For many years, research and development of medications for MS progressed slowly, with few significant milestones. However, in recent years, several novel mechanisms have accelerated progress and improved treatment for those diagnosed with MS. Of note, several of the earliest approved medications for MS are nearing the end of their original patent exclusivity, and it will be interesting to see how future MS guidelines are determined. As new medicines are studied and approved, more research is needed comparing these therapies to decide which will provide the most benefit.