Cerdelga (eliglustat) will become the first oral treatment that England’s National Health Service (NHS) makes available to patients with Gaucher disease.
At the moment, the only treatment the health service covers is enzyme replacement therapy administrated by intravenous infusion. It will begin making Cerdelga available later this year.
A medical advisory agency assured the health service’s use of Cerdelga when it reversed a preliminary recommendation against the therapy, which was developed by Sanofi Genzyme.
That agency, the National Institute of Health and Care Excellence, recommended that the health service make Cerdelga available as a first-line therapy for adults with type 1 Gaucher disease. The recommendation superseded a preliminary decision it made in March against the health service using Cerdelga.
The next step in adding Cerdelga to the health service menu will be for the agency, also known as NICE, to create guidelines for the drug’s use. They are expected before the end of June. The health service must make Cerdelga available within 90 days of the guidelines being published, which would mean no later than October.
‘’The news that NHS England will fund an oral treatment for type 1 Gaucher disease in England will be welcomed within the Gaucher community,” Tanya Collin-Histed, chief executive of the United Kingdom’s Gauchers Association, said in a press release.
“With this decision, patients in England, in consultation with their Gaucher Consultant, will be able to choose a treatment that best suits their needs,” she said. “Having the option of either oral or IV therapy available on the NHS will give many patients access to a new therapeutic opportunity.”
In contrast with standard care, Cerdelga does not replace enzymes that are abnormal or lacking. It is a substrate reduction therapy, meaning that it partially inhibits the enzyme responsible for producing glucosylceramide. An abnormal accumulation of glucosylceramide is a hallmark of Gaucher.
Two Phase 3 clinical trials, ENGAGE (NCT00891202) and ENCORE (NCT00943111), demonstrated that Cerdelga was effective and safe for patients with type 1 Gaucher. The key finding was that twice-a-day doses of Cerdelga were as good as enzyme replacement therapy in combatting the disease.
“Those living with the condition [Gaucher disease] in England are one step closer to gaining access to Cerdelga, the first NICE-recommended oral alternative to IV [intravenous] infusion therapy,” said Peter Kuiper, Sanofi Genzyme’s general manager for the United Kingdom and Ireland.
“Cerdelga is an established first-line treatment in many countries,” he said. “We hope that other UK national regulatory bodies will follow NICE’s example, allowing patients access to the same choice of treatment as those living in England and elsewhere in the world.”
Each of the countries in the United Kingdom has its own drug regulatory agency.