Today, the National Institute for Health and Care Excellence (NICE) announced that Cerdelga is recommended within its marketing authorisation for treating type 1 Gaucher disease, that is, for long-term treatment in adults who are cytochrome P450 2D6 poor, intermediate or extensive metabolisers. This recommendation means that the NHS must make Cerdelga available to patients in England, where the doctor responsible for their care thinks Cerdelga is the right treatment, within 3 months from today.
Prof Timothy Cox, Addenbrooke’s Hospital, Cambridge said “This is such positive news! The announcement by NICE is a long-awaited – but truly excellent - recommendation for British patients with Gaucher disease. After 15 years of clinical development, eliglustat received European marketing authorization in early 2015 for adult patients with type 1 Gaucher disease. With British patients participating in this massive trial programme, the news that the drug, (marketed as Cerdelga by Genzyme Sanofi), will be available for NHS patients, is wonderful to receive.”
“This is a potentially life changing moment for many patients in England, said Dan Brown, Chairman of the Gauchers Association. I would like to say thank you to Genzyme, on behalf of all of our members, for their continued commitment to the treatment of Gaucher disease. In this time of continued financial constraint within the NHS, huge credit must also go to all of those within the Gaucher community who worked so hard in supporting the NICE approval process to achieve such a positive outcome.”
Chief Executive, Tanya Collin-Histed said “The Association would like to say a huge thank you to the Gaucher community who completed a questionnaire that supported our patient submission for the NICE appraisal and to our two patient experts that took part in the appraisal process. Patient expert input is vital in this process, it is also a huge responsibility and therefore we are extremely grateful to you all for your support.”
More Details, written by Prof Timothy Cox;
NHS Gaucher centres should be able to prescribe the agent (where suitable) to adult patients who request it. Of course, there is a lot of organization to do - and the NHS has a statutory 90 days before the drug can be paid for. This would work out to be early September.
Each of the national treating centres will be busy making their own plans on how best to communicate with their patients.
Cerdelga is an orally active drug (most people will be prescribed two capsules a day; some; one) that is for most patients comparable to enzyme therapy in its effectiveness. There is an extensive safety profile now built up. While special testing is needed to guide dosing and to confirm that the drug will be suitable (for about 95% of people this is the case) patients should have this option very soon. There will be lots of questions that patients and families will have about the treatment and the centres will be working hard with the company to explain these.
Now after the first enzyme therapy 25 years ago, it is marvellous to see the options continuing to open up for patients: now with imiglucerase and velglucerase alfa, two safe and genetically engineered enzyme preparations, the first approved oral agent, miglustat, the field continues to change so much for the better. But even now, as members are aware, there is still more to do. However, this is an incredible boost to the cause of Gaucher medicine and will stimulate huge interest in continuing developments for all patients.