NICE rejects Biomarin’s £520K per year rare disease drug

The benefits of Biomarin’s Brineura for a very rare inherited condition do not justify its price of more than £520,000 per year, NICE has said in first draft guidance.

NICE had been assessing Brineura (cerliponase alfa) for children with neuronal ceroid lipofuscinosis type 2 (CLN2), a rare inherited condition affecting between one and six babies each year in the UK.

Also known as Batten disease, CLN2 is a progressive condition caused by the deficiency of the enzyme tripeptidyl peptidase 1.

This results in the abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.

Symptoms in children with CLN2 begin from around the age of two, and can then progress rapidly with the onset of seizures, decline in speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.

There is no cure or life-extending treatment for CLN2, other than symptom relief and supportive and palliative care.

Costing nearly £523,000 for a year’s treatment, based on list price of £20,107 for a 300mg pack, Brineura is an enzyme replacement therapy administered directly into the brain once a fortnight via a surgically implanted device. Lower doses are recommended for children under two years.

NICE noted that Brineura cannot cure CLN2 but said that it is effective in the short term at slowing rate of diseases progression.

But citing a lack long-term evidence about its effectiveness in stabilising the disease and preventing death, NICE’s Highly Specialised Technology committee said the therapy was not likely to be a cost-effective use of NHS resources.

However there is hope for those with the disease as BioMarin has said it will work with NICE to draw up a managed access agreement that could allow patients to access the drug until further longer-term clinical data is available.

This preliminary decision is open to consultation until 5 March.


In a separate final decision, NICE has recommended EUSA Pharma’s Fotivda (tivozanib) daily pill as a first-line treatment for advanced renal cell carcinoma, following an initial rejection by NICE.

As has often been the case, EUSA Pharma won backing from NICE after offering a confidential discount, in this case from a list price of more than £2000 for a three-week supply.

Based in the UK, EUSA Pharma has already got two new cancer drugs approved after spinning out of Jazz Pharmaceuticals in 2015.

Fotivda, a vascular endothelial growth factor receptor tyrosine kinase inhibitor (VEGFR-TKI) that has bettered Bayer’s rival Nexavar (sorafenib) in progression-free survival in a head-to-head trial.

The phase 3 TiVO-1 trial involving 517 patients with advanced renal cell carcinoma showed progression-free survival in patients treated with Fotivda was 11.9 months compared with 9.1 months in the overall population.

In treatment-naïve patients the progression-free survival on Fotivda was 12.7 months compared with 9.1 months on patients treated with Nexavar.

Only 14% of patients required a dose reduction due to adverse events compared with 43% on Nexavar, and fewer people on Fotivda experienced diarrhoea (23% vs 33%) and hand-foot syndrome (14% vs 54%).

The drug will also compete against Pfizer’s Sutent and Novartis’ Votrient in the first line indication.


NICE has recommended LEO Pharma’s Kyntheum (brodalumab) for regular NHS funding  in final guidance as a treatment for adults with severe plaque psoriasis.

The company got backing from NICE after offering a confidential discount to the NHS and the drug is expected to become available to the NHS next month.

AstraZeneca licensed rights to brodalumab in Europe to LEO Pharma after a link with suicidal ideation emerged in clinical trials. In the US, AZ licensed brodalumab to Valeant, which markets it under the brand name Siliq.

Kyntheum will now compete in a crowded market where new drugs such as Novartis’ Cosentyx and Eli Lilly’s Taltz are setting standards in care. Janssen also launched its new psoriasis drug Tremfya in the UK late last year.

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