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The profitability monitoring center in England rejected BioMarin's Brineura (Cerliponase alfa) therapy during the final surveillance.
The decision, which was published by the NICE Highly Technological Technology (HST) Committee, marks the end of a one-year negotiation between BioMarin and NHS England.
The disease affects only six children a year (50 in the UK are affected by this disease), but the price per patient, £ 500,000 ($ 651,100) per year, was considered unprofitable. absence of long-term efficacy data.
The committee initially agreed that Brineura had substantial short-term benefits in slowing the rate of progression of Batten's disease in the referral project, but stated that BioMarin was unable to set the price of treatment for a level that would have solved the problems identified in the NICE evaluation.
Meindert Boysen (below), director of the NICE Center for Health Technology Assessment, said: "The committee concluded that it was not possible within the NHS to manage complex elements of the confidential business arrangement proposed by the company.
"Once these elements are excluded, the cost-effectiveness estimates of cerliponase alfa are much higher than what NICE normally considers acceptable for highly specialized technologies. We therefore have no choice but to conclude that cerliponase alfa can not be recommended as a cost-effective use of NHS resources. "
Although Brineura is not a cure, it is an enzymatic replacement therapy administered directly into the brain via a permanently implanted surgical access device.
Results from the Phase 1 and 2 clinical trials show that people treated with Brineura reduced the decline in motor skills and language functions in children with Batten's disease.
Also known as CLN2, the disease is caused by tripeptidyl peptidase 1 enzyme deficiency. This results in abnormal storage of proteins and lipids in neurons and other cells, preventing them from functioning normally.
Symptoms begin from 2 years and can evolve rapidly with the onset of epileptic seizures, loss of speech, loss of mobility, involuntary muscle spasms, progressive dementia and visual impairment leading to blindness.
At present, no life-saving or curative treatment is available for CLN2 and clinical management is limited to symptom relief and supportive care.
Boysen added, "Given the tremendous burden this disease places on children with this disease, as well as their parents and caregivers, and the negative impact it can have on the quality of their lives, anything that can help improve treatment should be welcomed.
"We and NHS England have been very clear with the company on what would be necessary for us to recommend Cerliponase alfa. However, while there has been ample opportunity to come up with a viable solution, the company has unfortunately not been able to do so. "
Despite the slowdown in the UK, the drug was the third largest beneficiary of BioMarin in 2018, generating $ 31.3 million, attributed to new patients starting treatment after its product launch in mid-2017.
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