Siponimod (also known as BAF312 or Mayzent) is a tablet being developed for secondary progressive MS by Novartis Pharmaceuticals. It was licensed by the European Medicines Agency (EMA) on Monday 20 January 2020.
- Current phase of trial:
- Stage 3
How does siponimod work?
Siponimod works in a similar way to the licensed treatment fingolimod.
It traps certain types of immune cell (called B and T cells) in the body's lymph nodes. This stops them from getting into the brain and spinal cord, where they could cause damage to the protective myelin coating around the nerves.
How is siponimod taken?
Latest siponimod research
The EXPAND trial
This phase 3 trial involved 1,651 people with secondary progressive MS.
Top line results from the EXPAND trial were announced at a scientific conference in September 2016 and published in The Lancet journal in 2018.
Siponimod was found to reduce risk of disability progression by 21% compared with placebo. Only 26% of people on siponimod experienced a worsening of disability while on the trial, compared to 32% who took a placebo (dummy drug). Siponimod also significantly reduced the rate of brain atrophy (shrinkage) and the number of relapses people experienced.
Generally, siponimod was well tolerated, but like other DMTS, some people did experience side effects. These ranged from minor side effects like runny noses, to more serious conditions, like high blood pressure and low white blood cell count.
Results from a five year trial extension looking at long-term effects and safety were announced in April 2020. Data showed that people with secondary progressive MS who are continuously treated with siponimod experienced a lower risk of disability getting worse and cognitive decline, compared to patients who delayed siponimod treatment.
It’s still unclear whether siponimod can help people who've had secondary progressive MS for many years but the results of this trial are a positive step forward.
The BOLD trial
This phase 2 trial involved 188 people with relapsing MS and tested different doses of the drug. Siponimod was found to reduce relapse rates compared with placebo, and MRI scans also showed that siponimod reduced the number of brain lesions.
Long-term results were announced in July 2016. Novartis reported siponimod benefits were maintained at two years, particularly in people taking the higher doses of the drug (10 mg, 2 mg and 1.25 mg).
What are the side effects of siponimod?
Side effects reported in the phase 2 study included headache, slowing of heart rate, dizziness and nose and throat infections. In the EXPAND study side effects ranged from runny noses, to more serious conditions, like high blood pressure and low white blood cell count.
When is siponimod likely to be available?
In January 2020, the European Medicines Agency (EMA) licensed siponimod for people with active secondary progressive MS. But before it’s prescribed on the NHS it needs to be recommended by:
- the National Institute for Health and Clinical Excellence (NICE) in England and Wales
- the Scottish Medicines Consortium (SMC) in Scotland
In Northern Ireland, the NHS usually applies NICE recommendations.
In June 2020, NICE made a ‘provisional decision’ that siponimod should not be available. Although the SMC hasn’t yet decided, the NICE decision might have a knock-on effect on access across the UK.
We know this decision could have a negative impact on thousands of lives. We’re urging NICE to reconsider – to look again at the evidence and the reality of life with secondary progressive MS. Find out more and get involved in our feedback to NICE.