Shire has some good news to report. And after the unexpected FDA approval of a rival to its big-selling ADHD drug, Shire ($SHPGY) needed some good news. The company announced trial results showing its Gaucher disease drug Vpriv improved bone density in patients' spines, but Sanofi's ($SNY) Cerezyme didn't.
As Reuters reports, Gaucher disease is a genetic enzyme deficiency that can lead to organ damage and bone problems. The head-to-head Vpriv-vs.-Cerezyme trial was designed to determine the drugs' effects on Gaucher-related bone disease. Significant improvements in bone measurements were observed after 9 months of treatment in Vpriv patients, but not in Cerezyme patients.
"Many type 1 Gaucher disease patients experience bone abnormalities," Ari Zimran, a professor at Hadassah Medical School in Jerusalem, said in a statement. "These study results show that Vpriv is effective in treating selected markers of Gaucher-related bone disease, allowing these patients to achieve an important therapeutic goal quickly."
The data could help Vpriv distinguish itself in the increasingly crowded Gaucher-treatment world. Cerezyme was the only option for patients till Vpriv came along, and now, Pfizer ($PFE) and Protalix Biotherapeutics have won U.S. approval for their drug Elelyso. Only 10,000 patients are estimated to have the disorder worldwide, but these rare-disease drugs can command premium prices. As Reuters notes, Cerezyme was at one point the most expensive drug in the world, at $200,000 per year.
So, an edge in Gaucher disease could help offset generic erosion for Shire's Adderall XR drug. The company's stock tanked when news of the unexpected competition surfaced--so much so that analysts figure it's cheap enough for renewed takeover interest, Bloomberg reports.