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Daily Record
Daily Record
National
Hannah Carmichael

Advice published for three new medical treatments in Scotland

Three new medicines have had advice published for use in conditions including a rare and fatal genetic condition affecting young children and a form of leukaemia.

The Scottish Medicines Consortium (SMC) issued the advice for atidarsagene autotemcel (Libmeldy), venetoclax (Venclyxto) and upadacitinib (Rinvoq).

Libmeldy, a highly specialised stem cell treatment given as a single dose, can be used to treat children with a rare genetic condition called metachromatic leukodystrophy (MLD).

MLD causes damage to the nervous system, and children with the condition rapidly become unable to walk, talk, swallow, see and hear.

The condition is fatal, with children usually only living for five to eight years.

The use of Libmeldy could potentially allow children to develop physically to fully participate in everyday life and attend school.

However, the clinical evidence available is limited, and the cost in relation to the health benefits remains high.

Libmeldy will be made available for three years while information is gathered, after which the SMC will review the evidence and make a decision on routine availability in NHS Scotland.

Venclyxto was accepted for the treatment of acute myeloid leukaemia (AML), a cancer of the white blood cells which approximately 200 people in Scotland are diagnosed with every year.

It predominately affects older people, with more than four in 10 new UK cases being diagnosed in people aged 75 and over.

Used in conjunction with a chemotherapy medicine, venetoclax can be used in patients who have been newly diagnosed with AML and are unable to have intensive chemotherapy.

It is an oral once-daily treatment that can be taken at home, resulting in fewer hospital visits for clinically vulnerable patients.

It was found that the addition of venetoclax to current treatment may increase overall survival and reduce symptoms, thereby improving the wellbeing and quality of life of patients.

Upadacitinib (Rinvoq) was also accepted as an additional treatment option for adults and adolescents with moderate-to-severe atopic dermatitis, a chronic inflammatory skin condition.

The treatment is taken orally and may help patients better manage their care.

SMC chairman Mark MacGregor said: "Metachromatic leukodystrophy is a devastating hereditary disorder.

"The patient group representatives shared powerful personal evidence with the committee, describing the extent of disability the condition causes and its life changing impact on patients and families.

"Atidarsagene autotemcel will be available in NHS Scotland through the ultra-orphan pathway.

"This will allow patients with MLD to access treatment while more information on its clinical effectiveness is gained."

Mr MacGregor added: "The cost is, however, extremely high.

"It is essential that the pharmaceutical industry works to improve its efficiency in developing these life changing therapies if the costs are to be sustainable for health systems around the world."

Dominic Culligan, consultant haematologist and honorary senior lecturer at Aberdeen Royal Infirmary and the University of Aberdeen, said: "The new treatment option of venetoclax plus azacitidine is very welcome news for patients in Scotland.

"It is a significant advancement in the treatment of AML for patients not suitable for intensive chemotherapy and represents a step change in therapy for this aggressive, difficult-to-treat blood cancer."

Belinda Byrne, medical director at AbbVie UK, which is developing venetoclax alongside Roche, said: "We are delighted that the SMC has recommended venetoclax in combination with azacitidine as there has been an urgent need for effective new treatments for AML for many years.

"Routine availability of this combination for patients with this aggressive blood cancer could provide them with that all-important, and precious, time back with their families."

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