First drug approved for primary progressive MS
10th November 2017 - The first ever drug for primary progressive multiple sclerosis (PPMS) has been recommended for use in the UK.
However, it is only approved for people in the early stages of PPMS and a decision on funding has yet to be taken. There are still no treatment options for those who have had the condition longer.
Primary progressive MS
Multiple sclerosis is a neurological condition which affects the brain and/or spinal cord, causing problems with balance, sensation, vision and mobility. It occurs when the myelin sheath, the protective covering that surrounds nerves in the brain and spinal cord, is damaged.
Most people with MS will have a type known as relapsing and remitting MS (RRMS), meaning it comes and goes with gradual but not always complete recovery. A number of disease modifying drugs are available for this type of MS.
For around 10% of multiple sclerosis patients the disease is characterised by worsening symptoms from the beginning and there are no relapses/remissions. This is PPMS.
PPMS affects both men and women equally and is usually diagnosed in people in their 40s or 50s.
Until now there has been no available treatment for this type of MS.
The European Medicines Agency (EMA) has now recommended granting a marketing authorisation for ocrelizumab (brand name Ocrevus) for adults with early primary progressive multiple sclerosis.
In patients with this type of MS it was found to reduce 12 week disability progression by 24% compared with placebo (dummy treatment). After 120 weeks of treatment the drug also scored better on the time needed to walk 25 feet.
Ocrelizumab is taken as an intravenous infusion in an initial dose as split infusions 2 weeks apart, then further doses by infusion every 6 months.
It works by targeting a type of immune cell (called a B cell). This helps to reduce the immune response responsible for MS by stopping these cells from attacking and damaging myelin.
Data from the clinical trial in PPMS indicates that patients in the early stage of the disease benefit most from the drug.
It has also been recommended for use in people with RRMS.
In clinical trials the most common side effects were infusion-related reactions and infections.
Neoplasms - abnormal growth of tissues which can be benign or malignant - including several cases of breast cancer, were reported more frequently in those taking ocrelizumab.
What happens now?
The positive opinion by the EMA’s Committee for Medicinal Products for Human Use (CHMP) is the first step. The European Commission will now make a final decision on the licence within the next 3 months. After that the National Institute for Health and Care Excellence (NICE) will decide if ocrelizumab should be available on the NHS in England and Wales. Northern Ireland will decide whether or not to adopt the NICE decision shortly afterwards.