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Novartis to Get Expedited FDA Review of Secondary Progressive Multiple Sclerosis Drug

Phase 3 data shows siponimod has beneficial effects on disability, relapses and MRI disease activities (Image: Novartis)

~ Phase 3 data shows siponimod has beneficial effects on disability, relapses and MRI disease activities for patients with SPMS ~

Multiple sclerosis (MS) is a chronic, disabling disease of the central nervous system, affecting the brain, spinal cord and optic nerves. Estimates by the various medical and support organizations have determined historical prevalence to be about 2.3 million people worldwide, but this number appears to be low based on the most recent studies. That 2.3 million figure includes an older estimate of 400,000 US patients, but a study presented at the 2017 meeting of European Committee for Treatment & Research in Multiple Sclerosis (ECTRIMS), the world’s largest professional organisation dedicated to the understanding and treatment of MS, estimated that there were nearly one million people in the US living with MS. The current worldwide number may actually exceed four or five million people.

More than 80% of people with relapsing or remitting MS (RRMS), which is the most common form of the condition at diagnosis, go on to develop secondary progressive MS (SPMS). SPMS is a form of MS that leads to progressive, irreversible disability, such as the need for enhanced walking aids and wheelchairs, bladder dysfunction and cognitive decline, largely independent of relapses. According to a broadly cited 2008 study by Helen Tremlett, PhD, the Canada Research Chair in Neuroepidemiology and Multiple Sclerosis at the University of British Columbia, there is a gradual increase in the number of patients transitioning to SPMS as they get older, with about 25% of patients progressing by 10 years after initial onset, 50% of patients by 20 years and more than 75% by 30 years.

Novartis NVS announced today that both the FDA and European Medicines Agency (EMA) have accepted the company’s New Drug Application (NDA) and Marketing Authorization Application (MAA), respectively, for siponimod, the company’s candidate for the treatment of SPMS in adults. Novartis used a review voucher in order to expedite the review of siponimod in the US. The FDA is expected to pass ruling in March 2019, while the EMA’s action is expected in late 2019.

Siponimod is the first investigational medicine to show a significant delay in disability progression in typical SPMS patients. With siponimod, we underpin our strong commitment to the MS community by reimagining care for people whose lives have been considerably disrupted by this devastating illness. We are closely working with the FDA and EMA to ensure siponimod is available for patients as soon as possible.

– Paul Hudson, Chief Executive Officer, Novartis Pharmaceuticals.

Siponimod is an investigational, selective modulator of specific subtypes of the sphingosine-1-phosphate (S1P) receptor. Siponimod binds to the S1P1 sub-receptor on lymphocytes, which prevents them from entering the central nervous system of patients with multiple sclerosis. The drug also binds to the S1P5 sub-receptor on oligodendrocytes and astrocytes, having the potential to modulate damaging cell activity, and preclinical studies suggest that it may prevent synaptic neurodegeneration and promote remyelination in the central nervous system.

Data from a Phase 3 study of siponimod, for which more than 50% of patients relied on a walking aid at study initiation, showed that the drug reduced the risk of three-month confirmed disability progression by 21% vs placebo, which was the primary endpoint of the study. Siponimod also meaningfully delayed the risk of six-month confirmed disability progression by 26% vs placebo and demonstrated favorable outcomes in other relevant measures of MS disease activity and progression. Further analyses of the study showed that siponimod reduced the risk of disability progression largely disassociated from relapses: three-month progression by 14$ to 20%, and six-month progression by 29% to 33%. A previous Phase 2 study in patients with RRMS showed that siponimod significantly reduced the annualized rate of relapses over six months compared to placebo.

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DISCLOSURE: The author has no positions or any other beneficial interest in the companies mentioned in this article.

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