Sanofi's ($SNY) Genzyme snagged a 6-month priority review from the FDA for what could become the first wide-spread oral treatment for Gaucher disease, a rare genetic disorder that causes abnormal fat buildup in cells, enlarging organs in the body.
Genzyme's eliglustat, to be marketed under the name Cerdelga, is a twice-daily pill that inhibits the enzyme glucosylceramide synthase, reducing the production of glucosylceramide, which amasses in the cells to cause engorgement and organ growth in patients with Gaucher disease type 1, the most common form. There are fewer than 10,000 people with the disease worldwide, according to the company.
Currently, Gaucher treatments require extensive enzyme therapy. Genzyme's proposed oral option functions at the source of the fatty buildup. And, of course, a pill twice a day is a far more attractive option than undergoing intravenous injections. Only one other oral option, miglustat, exists but is used for a very small number of patients who cannot recieve enzyme therapy.
The FDA's priority review will allow the treatment to move through the regulatory process within 6 months instead of 10, Sanofi said Wednesday in a release. In late October, the European Medicines Agency validated a marketing authorization application for Cerdelga.
According to a Reuters report, analysts from Thomson Reuters Cortellis expect annual sales of the pricey drug to reach $157 million by 2018. Genzyme's injectable Gaucher treatment Cerezyme was once the most expensive in the world at more than $20,000 a year, Reuters reports. Other treatments on the market currently are Shire's ($SHPG) Vpriv and Pfizer's ($PFE) Elelyso.
"The acceptance of our applications for Cerdelga represents another important milestone in our commitment to understand and respond to the needs in the Gaucher community, providing more choice for the treatment of patients," said Genzyme CEO David Meeker in a statement.
With two positive Phase III studies behind it, Genzyme's clinical program for Cerdelga is the largest ever conducted in the treatment of Gaucher disease, according to the company.
- here's the release (PDF)
- and here's the Reuters report