Research News: New Drug Application Accepted, Treatment for Pediatric MS, and Conference Highlights

FDA Accepts Application for Cladribine Tablets

On July 30, 2018, EMD Serono, Inc. announced that the United States Food and Drug Administration (FDA) had accepted a New Drug Application (NDA) for Cladribine Tablets (an oral formulation of cladribine). This is an investigational treatment for relapsing forms of MS. Accepted by the European Commission in August 2017 and by Canada in December 2017, cladribine is currently approved for the treatment of relapsing MS in 38 countries under the trade name Mavenclad®.

In studies, cladribine has been shown to reduce disease activity in patients with relapsing MS, including disability progression, annualized relapse rate, and MRI activity. Potential adverse events include lymphopenia, a condition that causes abnormally low counts of white blood cells that play a role in fighting infection, and herpes zoster.

According to MSAA’s MS Research Update 2018, “Cladribine selectively targets the immune system’s B cells and T cells, leading to depletion of those cells This is followed by a distinct pattern of ‘reconstitution,’ as new cells and T cells are produced. The medication has an interesting dosing regimen, with two annual courses given for a maximum of 20 days over two years. Its developers note that this approach avoids continuous suppression of the immune system.” Following these first two years, no treatment is needed for Years 3 and 4.

FDA Accepts Gilenya for Pediatric MS

On May 11, 2018, Novartis announced that Gilenya® (fingolimod) was approved by the United States Food and Drug Administration (FDA) for the treatment of children and adolescents, ages 10 through 17, with relapsing multiple sclerosis (MS). This is the first disease-modifying therapy (DMT) to be approved for this form of the disease in this age group.

Known as “pediatric MS,” nearly all (98 percent) of those diagnosed have the relapsing form of the disease. Children experience approximately two-to-three times as many relapses as an individual with adult-onset MS. Prior to this approval, all 15 disease-modifying therapies (DMTs) for MS had only been approved for adults; no treatment had been approved specifically for children and adolescents with MS.

The FDA’s approval of Gilenya was based on the results from the Phase III PARADIGMS study. This clinical trial evaluated the safety and effectiveness of Gilenya versus Avonex® (interferon beta-1a) in children and adolescents (ages 10 through 17) with relapsing MS. PARADIGMS is the first study to compare two MS therapies, approved for adults, in children and adolescents with MS. Enrolling 215 young individuals with MS, this study was conducted at 87 sites in more than 25 countries. PARADIGMS was a double-blind, randomized, multi-center Phase III study with a duration of up to two years, followed by a five-year, open-label extension phase.

Study participants were either given Avonex, a once-weekly intramuscular injection, or Gilenya, an oral pill taken once daily. Compared to the Avonex group, those taking Gilenya experienced an 82-percent reduction in their annualized relapse rate during a period of up to two years. Side effects and adverse events were similar to those seen in other clinical trials with adults.

For more information on Gilenya for treating pediatric MS, individuals may visit, or call 800-GILENYA (800-445-3692) to speak with a member of Novartis’ support team.

Research Highlights from the AAN and CMSC 2018 Annual Meetings

The results of numerous significant studies were reported at the Annual Meeting of the American Academy of Neurology (AAN), held in Los Angeles in April, and the Annual Meeting of the Consortium of Multiple Sclerosis Centers (CMSC), which took place in Nashville in May. Posted on July 6, 2018, MSAA provided a summary of some of the most interesting findings from those conferences.

Topics in this article include: updates on experimental disease-modifying therapies (DMTs); continuing studies on the effectiveness and safety of approved DMTs; comparative analysis of approved DMTs; new diagnostic testing; strategies for wellness; and hot topics in MS. This latter section includes information on stem cell transplant, gut microbiome, medical marijuana, and more. To read the full summary, please see “Research Highlights from the AAN and CMSC 2018 Annual Meetings.” For individuals without internet access, please call MSAA at (800) 532-7667 to request a printed copy.

To read all of MSAA’s latest articles online, please visit our website at and go to “Latest News.” For general information or to speak with a trained Client Services Specialist, please call MSAA’s Helpline at (800) 532-7667, extension 154. Questions to MSAA’s Client Services department may also be emailed to

Written by Susan Wells Courtney, MSAA Senior Writer

Reviewed by Dr. Jack Burks, MSAA Chief Medical Consultant