Advice published for three new medical treatments in Scotland

11.04.2022 - 22:37 / dailyrecord.co.uk

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.

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