NICE recommends Sanofi’s Gaucher treatment

The cost effectiveness body published final guidance under its Highly Specialised Technologies scheme recommending Cerdelga for treating type 1 disease.

Genzyme had offered a further price cut on top of what was already on the table, although details of the discount it provides are confidential for commercial reasons.

But NICE said the improved patient access scheme means Cerdelga is now cheaper than the current standard NHS treatment for the condition, which is enzyme replacement therapy such as Genzyme’s own Cerezyme (imiglucerase).

Information from NICE shows that the list price of Cerdelga is £342.23 per capsule, bringing the total cost to around £250,000 per year on average.

Gaucher is a genetic disease where glucocerebroside accumulates in cells and certain organs, with symptoms including fatigue, anaemia, low blood platelet count and enlargement of the liver and spleen.

It is caused by deficiency of the enzyme glucocerebrosidase, which usually breaks down glucocerebroside.

While orphan drugs such as Cerdelga offer potent therapies for rare diseases, prices can be eye-watering.

In February, NICE rejected Alexion’s Kanuma for the ultra-rare disease LAL-D because of its £492,000 per year price tag.

In the same month it also rejected Alexion’s Strensiq for paediatric-onset hypophosphatasia, priced at £366,000 per year.

The European orphan drug patient group, Eurordis, has called for pharma to develop more rare disease drugs, but at a lower price.

Novartis and Pfizer drugs recommended for NETs

In separate guidance, NICE also said it was recommending regular NHS funding for treatments for neuroendocrine tumours (NETs) from Novartis and Pfizer.

Pfizer’s Sutent (sunitinib) had been reimbursed by the Cancer Drugs Fund until new arrangements were introduced last year.

Novartis’ Afinitor (everolimus) had been dropped from the CDF but was being reconsidered after the manufacturer offered a confidential discount.

In its final guidance, NICE said both drugs are recommended as an option for pancreatic NETs, with Afinitor recommended for those with NETs of gastrointestinal or lung origin where their disease has progressed.