NHS Scotland: Blood cancer and migraine treatments among medicines approved for use

New treatments for migraines and blood cancer are among the latest batch of medicines to have been approved for use in Scotland.

The Scottish Medicines Consortium (SMC), which advises on newly-licensed medicines for use by NHS Scotland, approved a total of six new treatments for a range of conditions.

Two gene therapy treatments were approved but will be monitored for three years before a review to decide whether to make them more widely available.

Adults who suffer at least four migraines per month were offered hope of “improved symptoms and quality of life” using oral treatment Rimegepant (Vydura).

One of the most common types of blood cancer, previously untreated chronic lymphocytic leukaemia (CLL), will be targeted using Ibrutinib (Imbruvica) after it was accepted for use together with Venetoclax (Venclyxto).

Other patients suffering rare blood cancer but who cannot have stem cell transplants to treat newly-diagnosed myeloma, will be offered hope from Daratumumab (Darzalex) after it was accepted as part of a combination.

Both blood cancer treatments were welcomed as “valuable treatment options” by Dr Scott Muir, chair of the SMC.

Adults suffering from life-threatening condition hereditary transthyretin-mediated amyloidosis, which can cause heart and neurological problems, will be able to access Vutrisiran (Amvuttra) after it was also accepted.

The first drug ever to target the rare genetic condition acid sphingomyelinase deficiency (ASMD), also known as Niemann-Pick disease, will be made available for three years before being reviewed.

New drug Olipudase alfa (Xenpozyme) was approved to target the underlying causes of ASMD, which can affect the spleen, lungs, liver and blood, but it will be monitored for three years before a decision is made to make it routine.

A pioneering gene therapy treatment was approved for the first time to treat severe aromatic L-amino acid decarboxylase (AADC) deficiency – but only for three years before the drug will be reviewed.

The genetic condition affects the nervous system and causes developmental delays, muscle weakness, movement disorders and intellectual disability, but is hoped to be treated with Eladocagene exuparvovec (Upstaza).

SMC chair Dr Scott Muir said: “We are very pleased to be able to accept these medicines for use by NHS Scotland.

“Rimegepant (Vydura) offers an oral treatment option which may help improve symptoms and quality of life for those affected by migraines.

“We know from the patient and clinician engagement meetings that Daratumumab and Ibrutinib are considered valuable treatment options so we are pleased to be able to accept these medicines for use in people with rare blood cancers.”

Dr Sophie Castell, chief executive at blood cancer charity Myeloma UK, welcomed the news about Daratumumab as part of a combination treatment.

She said: “Approximately two-thirds of newly-diagnosed myeloma patients are not eligible for a transplant, and now, at long last, they’ll be able to benefit from a life-extending treatment that could give them five precious years with their loved ones.

“As the first nation to approve this treatment on the NHS, Scotland is truly leading the way in bridging the gap in survival between people who are eligible for a stem cell transplant and those who are not.”