Long-term Gilenya Treatment Safe and Effective for Relapsing MS Patients, Phase 3 Trial Shows

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Long-term treatment with Gilenya (fingolimod) in patients with relapsing forms of multiple sclerosis (MS) is safe and effective, results from a Phase 3 trial show.

Trial findings were reported in the study, “Extended treatment with fingolimod for relapsing multiple sclerosis: the 14-year LONGTERMS study results,” published in the journal Therapeutic Advances in Neurological Disorders.

MS is an autoimmune disease that affects the central nervous system (consisting of the brain and spinal cord), and given its chronic nature, “patients starting therapy should expect ongoing lifelong treatment to control disease activity and reduce the risk of disability accrual,” the researchers wrote.

“However, data on the safety and efficacy of disease-modifying therapies (DMTs) beyond 5–7 years of therapy are limited, leaving an important knowledge gap in a patient population that may have to undergo several decades or more of therapy,” they added.

The multicenter, open-label Phase 3 LONGTERMS (NCT01201356) trial was designed to assess the long-term safety and efficacy of Gilenya in patients with relapsing forms of MS.

Developed by Novartis, Gilenya was the first oral DMT to be approved by the U.S. Food and Drug Administration and the European Commission for the treatment of adults and children with relapsing-remitting multiple sclerosis (RRMS).

The medication works by preventing immune cells from reaching the brain and spinal cord, reducing inflammation, disease progression, and the risk of MS relapses. 

LONGTERMS enrolled 4,086 adults with relapsing MS who had previously participated in other clinical trials testing Gilenya — FREEDOMS (NCT00289978), FREEDOMS II (NCT00355134), and TRANSFORMS (NCT00340834) — and who had been exposed to the medication for more than a decade.

During the trial, all participants took 0.5 mg of Gilenya orally once a day, regardless of the treatment they had been assigned in previous trials.

The study’s main goal was to assess the long-term safety and tolerability of Gilenya by evaluating the incidence and severity of adverse events related to the treatment.

Secondary goals included assessing the impact of treatment on the number of annual MS relapses (annualized relapse rate, or ARR), and on the patients’ degree of disability, measured by the Expanded Disability Status Scale (EDSS) and by six-month confirmed disability progression.

Of the 4,086 patients initially enrolled in the study, 3,480 (85.2%) completed the trial. Patients with a median age of 38 years received Gilenya from 75 up to 4,777 days (from two and half months up to 13 years).

Results showed that most study participants (85.5%) reported at least one adverse event, the most common being upper respiratory tract infections (17.3%), headaches (13.3%), high blood pressure (11.0%), and low white blood cell counts (10.7%).

The most frequent serious adverse events, which were experienced by 12.6% of the study participants, were basal cell carcinoma and MS relapses, with a frequency of 0.9% each.

Secondary efficacy assessments revealed the ARR dropped from 0.22 within the first two years of treatment with Gilenya to 0.17 within the first decade. Moreover, after a decade of treatment with Gilenya, 45.5% of the patients remained free of relapses, and 63.2% showed no signs of disability worsening.

“In conclusion, these results suggest that continuous administration of fingolimod for 10 years or more in patients with [relapsing MS] is associated with sustained benefits for control of disease activity and disability progression without emerging safety concerns,” the researchers wrote.