Novartis Drug Fails to Reduce Disability in Primary Progressive Multiple Sclerosis in Trial

In a phase 3 clinical trial, Novartis's drug Gilenya (fingolimod) did not reduce disability in patients with primary progressive multiple sclerosis.

In a Phase 3 trial, Novartis’ drug fingolimod (Gilenya), designed to treat primary progressive multiple sclerosis (PPMS), did not demonstrate any differences from placebo, according to the company.

In the trial, researchers studied the effects of fingolimod and placebo on 970 PPMS patients aged 25-69 years across 18 countries. Patients were treated for at least 3 years. Researchers expected to evaluate disability progression based on a composite measure of Expanded Disability Status Scale (EDSS), assessment of upper limb function, and walking speed. Researchers hypothesized fingolimod would enter the central nervous system (CNS) and interact with damage-causing cells residing inside, similar to other forms of multiple sclerosis (MS). Instead, data showed that in PPMS, fingolimod was not as efficacious and that relapsing MS forms have different mechanisms.

The drug is designed to prevent cells that cause focal inflammation from reaching the brain in addition to reducing the damage caused by harmful cells in the CNS. Combating both of these effects can help reduce the disease’s impact on a patient’s physical and cognitive functions.

“We understand this news is very disappointing for those affected by PPMS and involved in its management,” Vasant Narasimhan, Global Head of Development at Novartis Pharmaceuticals said in a press release. “While PPMS is a focus of the MS community, relatively little is known about the disease so finding effective treatments remains a challenge. We will actively work with the MS community to review and analyze the INFORMS results to help increase the understanding of this devastating disease. Fingolimod revolutionized the treatment of relapsing MS as the first oral disease-modifying therapy. We remain strongly committed to continuing to research new treatment options for patients with MS and other neurological conditions.”

Currently, there is no approved treatment for PPMS, which is different from other types of MS. PPMS effects about 10 percent of the total MS population, which is about 2.3 million patients globally. However, fingolimod is approved in the US for first-line treatment of relapsing MS in adults. It is marketed as the only disease-modifying therapy (DMT) to impact the development of MS across 4 prongs: relapses, MRI lesions, brain shrinkage by volume, and disability progression. In MS, the chance of “no evidence of disease activity” is 4 times greater in relapsing MS patients treated with fingolimod. Novartis noted they believe they will soon identify and develop treatment options for PPMS.