Two medicines accepted for use by NHS Scotland

The Scottish Medicines Consortium has approved NHS funding for treatments for blood cancer and a rare muscle disorder.

AbbVie's Venetoclax (venetoclax) was accepted for the treatment of certain patients with previously untreated chronic lymphocytic leukaemia (CLL).

The decision allows doctors to prescribe the drug in combination with obinutuzumab as a first-line treatment for CLL patients without del (17p)/TP53 mutation when they are not fit to receive FCR (fludarabine, cyclophosphamide and rituximab) chemo-immunotherapy, and also for those with higher risk disease due to the presence of del (17p)/TP53 mutation.

The medicine was considered 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 highlighted that CLL is an incurable, relapsing blood cancer that tends to affect older adults.

Venetoclax, given alongside obinutuzumab, offers a chemotherapy-free, 12-month fixed-duration treatment that has been demonstrated to improve outcomes, allowing patients to live significantly longer without their disease progressing, compared to those receiving a commonly used chemo-immunotherapy, according to AbbVie.

Also, as an oral therapy that can be administered at home it could allow some patients to continue work or engage more fully in family life, the SMC noted.

“The SMC’s decision to make the venetoclax plus obinutuzumab combination treatment available within NHS Scotland to this group of patients is welcome news to the CLL patient community,” said Marc Auckland, CLL patient and chair of the CLL Support Association.

“This new fixed one-year treatment offers patients the opportunity of prolonged remission off treatment, which can help them to stay in work and participate in family and social activities. This can have immense psychological benefits.”

Also accepted through PACE following a resubmission was Lupin Healthcare's Namuscla (mexiletine) for the treatment of symptoms of myotonia (muscle stiffness) in patients with non-dystrophic myotonic (NDM) disorders.

NDM is a group of muscle disorders in which muscles are slow to relax after movement.

In the PACE meeting, patient group representatives and clinicians spoke of how the main symptoms of this rare condition, including muscle stiffness, pain, weakness and fatigue, can have a significant impact on patients’ daily lives. The condition also has an emotional impact on both patients and their carers, limiting their ability to work and socialise.

This formulation of mexiletine is the first medicine to be licensed for the symptomatic treatment of myotonia in patients with NDM. According to the SMC, the drug may improve symptoms which could allow some patients to be independent, relieving the anxiety and distress associated with this condition.

On the downside, Daiichi Sankyo's Nilemdo (bempedoic acid), a medicine that can be used to lower levels of cholesterol in the blood, was not accepted “because the evidence provided by the company was not strong enough to satisfy the committee that it is a good use of NHS resources,” the cost watchdog stressed.