Sanofi's ($SNY) rare ailment-focused Genzyme unit won FDA approval for a new Gaucher disease treatment, providing an oral alternative to its own trailblazing intravenous therapy.
The drug, developed as eliglustat, is designed to treat the symptoms of Gaucher, which stems from a deficiency of the enzyme glucocerebrosidase and leads to fatty buildups in the spleen and liver. Unlike Genzyme's Cerezyme, which replaces the missing component, the new drug is designed to inhibit the metabolic process that helps the deposits form, eschewing injections in favor of a twice-a-day pill.
In its late-stage program, the treatment successfully stabilized the disease across two trials: one placebo-controlled study on treatment-naive patients and another demonstrating its noninferiority to enzyme replacement.
Sanofi plans to launch its drug under the brand name Cerdelga within a month, looking to siphon patients away from Cerezyme--which accounts for about $916 million in annual sales--and competing injections from Shire ($SHPG) and Pfizer ($PFE). Sanofi's new Gaucher contender is likely to bring in around $749 million a year by 2020, according to Bloomberg's analysis, but how much of that will come out of Genzyme's own pocket remains to be seen.
The company said it's still determining just what its new rare disease drug will cost, but noted that it will be in the same ballpark as Cerezyme, which goes for about $300,000 a year in the U.S.
Cerdelga's approval caps a 15-year effort by Genzyme to develop an oral Gaucher treatment, and its launch will nearly coincide with the anniversary of its forebear, CEO David Meeker noted.
"More than 20 years ago, Genzyme introduced the world's first treatment for Gaucher disease," Meeker said in a statement. "We are proud to build on this legacy and continue to improve Gaucher patients' lives through ongoing research and new therapies."
The company estimates that just 10,000 patients around the world suffer from the disease, with about 6,000 living in the U.S.
- read Sanofi's statement
- here's the FDA's release
- check out Bloomberg's story