April 2021 decisions news release

Advice on four new medicines for use in conditions including multiple myeloma and severe migraine has today (Monday 12 April) been published by the Scottish Medicines Consortium. A new medicine for Duchenne Muscular Dystrophy has now also been included in the ultra-orphan pathway.

An initial assessment report has been published for ataluren (Translarna), which can be used to treat the genetic disease Duchenne Muscular Dystrophy. This medicine has been assessed through the ultra-orphan pathway for medicines which treat very rare conditions. Duchenne Muscular Dystrophy is a rare, progressive muscle-wasting condition that affects mostly boys, gradually causing weakness and loss of muscle function. Current treatment options focus on the management of symptoms rather than addressing the underlying genetic cause. Ataluren can be used to treat the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene. It may allow affected children to retain the ability to walk for longer, potentially leading to increased independence. The submitting pharmaceutical company is now required to provide a plan detailing how further data on the effects of the medicine, including those on the patient and carer lived experience, will be collected. Ataluren will then be available through NHSScotland for three years while the information is gathered. Following this, SMC will review the evidence and make a decision on routine availability in NHSScotland. Further information on the ultra-orphan approach can be found in Scottish Government guidance.

Isatuximab (Sarclisa) was accepted for the treatment of relapsed or refractory multiple myeloma (a cancer of the bone marrow). The medicine, which is used in combination with two other medicines, pomalidomide and dexamethasone, was accepted following consideration through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and rare conditions. In the PACE meeting, participants spoke of how patients can suffer debilitating symptoms including recurrent infections, anaemia, fatigue and severe bone pain and fractures. Patients who have had multiple relapses may also carry the burden of side-effects remaining after previous treatments. As the disease progresses, remissions can be shorter and more difficult to achieve. Isatuximab increases progression free survival and has an acceptable side effect profile, providing patients with another treatment option.

Dupilumab (Dupixent) was accepted for the treatment of a subgroup of patients with severe asthma. People with severe asthma do not respond fully to standard treatment and require more intensive therapies to control their symptoms. Intensive therapies include oral corticosteroids, which can have toxic side effects such as osteoporosis and diabetes. Dupilumab offers an additional treatment option for those with this form of asthma.

Dapagliflozin (Forxiga) was accepted for the treatment of heart failure in patients who have symptoms of the disease and a reduced ejection fraction (where the proportion of blood leaving the heart is less than the body needs). Patients with heart failure often experience breathlessness, fatigue and oedema (fluid retention). Dapagliflozin offers another treatment option for those patients who remain symptomatic despite the optimisation of their current treatment, and may help reduce breathlessness and fatigue improving their quality of life and potentially lowering the risk of early death.

Also accepted was galcanezumab (Emgality) for the prevention of migraine in adults who have at least four migraine days per month. Galcanezumab is a preventative treatment specifically designed for migraine and provides another treatment option which may improve symptoms and quality of life.

SMC Chairman Mark MacGregor said:

“Ataluren will now move to the next stage of the ultra-orphan pathway. This will allow some patients with Duchenne Muscular Dystrophy to access treatment while more information on its clinical effectiveness is gained. Scottish Government will announce when this medicine will be available for prescribing in NHSScotland.”

“The committee is pleased to be able to accept these four medicines for use by NHSScotland.”

“From the evidence given by patients and clinicians in the PACE meeting, we know that our decision on isatuxumab will provide a valued additional treatment option for those with multiple myeloma.”

“For patients with severe asthma with type 2 inflammation, our decision on dupilumab offers an alternative to current treatment options.”

“Our decision on dapagliflozin offers the possibility of lowering the risk of early death and an improved quality of life for patients with heart failure.”
“Galcanezumab offers another treatment option which may help improve symptoms and quality of life for those affected by regular migraines.”

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