Multiple sclerosis (MS) is a disease of the central nervous system (CNS). It occurs when myelin, which protects nerve fibres so messages can travel smoothly and quickly between the brain and the rest of the body, for an unknown reason, is treated by the immune system as a foreign object and gets damaged.
This disrupts messages travelling along these nerve fibres, causing a range of disabling, but often invisible, symptoms dependent on the part of the CNS affected.
To raise awareness of this debilitating disease, which affects approximately 2.5 million people worldwide, on 30 May every year the MS International Federation coordinates World MS Day. This year the campaign focuses on MS’s unseen impact on patients’ quality of life.
Roche’s Ocrevus: journey to NHS use
The core way MS is managed and treated is using so-called disease modifying drugs (DMDs). Other medications are used to help manage symptoms resulting from the condition.
DMDs have traditionally been used to reduce the number or severity of relapses, a sudden episode of new symptoms or worsening of symptoms, known as relapsing forms of MS (RMS).
However, with the approval of Roche’s DMD Ocrevus (ocrelizumab) for primary progressive MS (PPMS), where patients experience a steady worsening of symptoms over time, as well as for (RMS), the capabilities of DMDs have expanded. PPMS represents around 10% of all MS cases.
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Ocrevus was approved for PPMS by the US Food and Drug Administration (FDA) in 2017; the European Medicines Agency (EMA) followed suit in January 2018.
Although this meant Ocrevus was now approved across Europe, in September 2018 the UK’s healthcare regulator the National Institute of Health and Care Excellence (NICE) decided to only recommend the drug for RMS.
Despite NICE acknowledging that Ocrevus slowed the worsening of disability in PPMS patients, it concluded that the benefits of the drug in this patient population did not represent value for money for the NHS.
The regulator reversed this decision in May this year following a commercial agreement between Roche and NHS England over the price of Ocrevus spurred on by a strong patient advocacy campaign. However, there are some restrictions in NICE’s recommendation regarding who can use Ocrevus, including having PPMS symptoms for 15 years or less, ability to walk 20 metres or more and evidence of MS activity on MRI scans.
Responses to NICE’s recommendation of Ocrevus
NICE’s move was greeted with huge optimism by MS charities and organisations working in the UK. MS Society director of external affairs Genevieve Edwards said: “This is a landmark moment and an incredible victory for the more than 21,000 of us who helped overturn this result. We now want to see everyone who could benefit from ocrelizumab being able to access it, with increased support for MS services to make sure this happens.
“Right now however there isn’t enough evidence to show ocrelizumab can work for everyone, and we know the restrictions will be a massive blow for those who still don’t have any options.
“We’re driving research to find more and better treatments, and calling for drug trials to more fully address the needs of everyone with MS, until the day we are able to stop it in its tracks.”
MS Trust chief executive David Martin commented: “This is very welcome news. We commend the willingness of all three parties to find a solution which enables people with early, inflammatory primary progressive MS to access a treatment which will allow them to continue working and remain independent for longer.
“But we know this is just the start. More treatments for progressive MS are still desperately needed, and we will continue to fight to ensure everyone with MS can access the treatments they need.”
Treatments in the pipeline for PPMS
One drug currently in Phase II development for PPMS, as well as secondary progressive MS (SPMS), is MediciNova’s ibudilast (MN-166); drug has the FDA’s fast track designation.
Ibudilast works by supressing three cytokines known to promote inflammation.
The company began the Phase IIb SPRINT-MS trial of the drug in the two types of progressive MS in 2013, supported by a grant from the US National Institutes of Health.
In results from subgroup analysis from the trial published in April, the drug was most effective in the non-relapsing group of secondary progressive MS patients where there was a 46% risk reduction compared to placebo. The PPMS group saw a 29% risk reduction.
Other analysis noted by the MS Trust showed that ibudilast significantly slowed brain atrophy in PPMS, but this was not seen in SPMS.
MediciNova is going to use this subgroup analysis to design its trial design for a Phase III programme in progressive MS; it is currently unclear if all progressive MS subgroups will remain included in the next trial.
Another drug being trialled for both PPMS and SPMS is Ab Science’s tyrosine kinase inhibitor mastinib (AB1010), which is already in Phase III.
Ab Science’s Phase III trial of mastinib is ongoing, and is expected to be completed by the end of 2019; 665 patients are enrolled and participating in the study.
In January 2018, an Independent Data Monitoring Committee recommended that the study could continue without the need for sample size re-estimation.
The primary endpoint of the study is change over 96 weeks in Expanded Disability Status Scale (EDSS). Ab Science’s interim analysis for the trial is positive.