Genzyme's recently approved orphan drug, Cerdelga, will be priced at $310,250 a year

Genzyme Prices Gaucher Disease Drug at $310,250 a Year

In Market Access, Supply Chain & Manufacturing by Cecile

Genzyme’s recently approved orphan drug, Cerdelga, will be priced at $310,250 a year

The Masachusetts-based biotech company Genzyme, owned by pharmaceutical giant Sanofi, will be pricing its rare disease drug for the treatment of Gaucher’s disease at $310,250 a year.

The US Food and Drug Administration approved the drug and granted it orphan drug status after it had shown positive results from its final phase III trials. Because the drug has been granted orphan drug status, Genzyme will be able to market the drug exclusively for 7 years, and will be able to apply for tax breaks.

This is not the first drug that Genzyme has developed for Gaucher’s disease. Indeed, Cerdelga may replace Genzyme’s Cerezyme drug, which is an enzyme replacement therapy that is required to be administered every two weeks via a blood infusion. In contrast, Cerdelga is a pill that only needs to be taken twice a day. Cerdelga is slightly more expensive than Cerezyme, which costs $300,000. Interestingly, the costs associated with manufacturing the Cerdelga capsule are less than the costs associated with producing the Cerezyme treatment.

Gaucher’s disease is a lipid storage disease that causes fat (in the form of sphingolipids) to accumulate in the organs. Cerdelga is able to stop sphingolipids from being stored in cells in the first place, whereas Cerezyme breaks down fat deposits that have already accumulated in cells.

This new treatment will have an immensely positive impact on the live’s of patients living with Gaucher’s disease, but unless the price of the drug is subsidised by government health care programmes, the expensive price tag on the drug may cause it to be out of reach for most patients.

Read more about it here: Genzyme Corporation’s New Rare Disease Pill Will Cost Patients $310,250 A Year