Four new medicines cleared for use on NHS Scotland

Tesaro UK’s Zeluja (niraparib) was accepted for use for advanced ovarian cancer, but only in patients who do not carry the BRCA mutation.

The drug won European approval in November last year as the first once-daily, oral poly (ADP-ribose) polymerase (PARP) 1/2 inhibitor cleared for ovarian cancer patients regardless of BRCA mutation status.

In clinical trials, the treatment cut the risk of disease progression or death by 73 percent in patients with germline BRCA mutations and by 55 percent in those without germline BRCA mutations, but the SMC highlighted that there are currently fewer satisfactory alternative treatments for the latter group of patients.

Elsewhere, Roche’s Alecensa (alectinib) was accepted for the treatment of a rare type of advanced lung cancer called ALK-positive non-small cell lung cancer.

The SMC noted that patients are often diagnosed when the disease is at a late stage and treatment options are limited, and that Alecensa "offers the benefit of an increase in the time to disease progression compared with existing therapy".

According to Roche, Alecensa has been shown to improve outcomes compared to current standard of care, including decreasing the risk of disease progression or death by more than half, and by reducing the risk of tumours in the central nervous system by 84 percent.

Swedish Orphan’s Ravicti (glycerol phenylbutyrate) was accepted for the long-term treatment of urea cycle disorders (UCDs) in adults and children from the age of two months.

A UCD is a rare, inherited genetic condition where patients are unable to get rid of waste nitrogen from their bodies. This then accumulates in the form of ammonia and may lead to irreversible brain damage, coma and/or death if untreated.

Ravicti reduces levels of ammonia in the body, offering an alternative to current treatment, "which is often difficult for patients to adhere to, particularly for children, due to an unpleasant taste and the high number of tablets required," the cost watchdog said.

Pharming’s Ruconest (conestat alfa) was accepted for the treatment of patients with the rare condition acute hereditary angioedema, which can cause painful and potentially dangerous attacks of swelling anywhere in the body.

The drug is a plasma-free C1-esterase inhibitor (C1-INH) shown to help relieve symptoms of HAE attacks, offering an additional treatment option for patients.

On the downside, the committee rejected NHS funding for BioMarin’s Kuvan (sapropterin) for phenylketonuria (PKU), a rare genetic condition where patients are unable to process the amino acid phenylalanine from dietary protein, because of "uncertainties in the company’s evidence about both the long-term benefits of the medicine and its cost effectiveness".

The committee was also unable to accept Vifor Pharma’s Veltassa (patiromer) for the treatment of hyperkalaemia, a condition in which potassium levels in the blood are raised. Evidence provided by the company about the cost effectiveness of the medicine "was not strong enough to justify accepting it for use by NHS Scotland," the SMC noted.

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