Today, the SMC announced that Cerdelga is recommended within its marketing authorisation for treating type I Gaucher disease, that is, for long-term treatment in adults who are cytochrome CYP2D6 poor, intermediate or extensive metabolisers. This recommendation means that the SMC must make Cerdelga available to patients in Scotland, where the doctor responsible for their care thinks Cerdelga is the right treatment.
Chief Executive, Tanya Collin-Histed said "The Association is delighted with the outcome, which will give patients a choice of treatment in consultation with their treating doctor". The Association represented Gaucher patients at two SMC meetings in October and November where they presented evidence about the impact of living with Gaucher disease and the challenges that regular infusions of Enzyme Replacement Therapy have on some patients. This decision comes seven months after Cerdelga was approved by NICE for patients living in England. Click here to read the full SMC report