Shire is pouncing on the big opening provided by rival Genzyme - which is facing ongoing manufacturing issues - to establish an early marketing foothold for its investigational enzyme replacement therapy, velaglucerase, for treating Type 1 Gaucher disease.
The company announced on Nov. 24 that it submitted a marketing authorization application for velaglucerase to the European Medicines Agency and has been granted an accelerated, 150-day review, given the global supply shortage of Genzyme's Cerezyme (imiglucerase).
Cerezyme is the only drug currently approved for the treatment of Gaucher disease, and an ongoing shortage of the drug has left some patients without medication for the rare genetic disorder that can result in bone pain, fractures and anemia.
Genzyme has been facing manufacturing delays for several of its key drugs since the summer as a result of contamination issues at its Allston, Mass. manufacturing facility. It has been a lingering cloud for the biotech, which is continuing to face challenges and recently announced another temporary shut down of the plant ("The Pink Sheet" DAILY, Nov. 13, 2009).
Lack of availability of an important treatment like Cerezyme has left an opening for competitors to get to the market sooner and to develop loyal patient relationships. Shire filed an NDA for velaglucerase in the U.S. in August, where the application was granted a six-month priority review. The FDA action date for the application is Feb. 28.
Additionally, some patients are already being treated with velaglucerase in the U.S. and Europe through special access programs. In the U.S., velaglucerase has been made available to patients since September under a treatment IND. Still, this head start will do little good if no drug is available. The challenge for Shire has been meeting demand, given that the company hadn't anticipated launching the drug until next year.
For now, Shire has predicted it will be able to treat 300 to 600 patients this year, which represents only a small fraction of the 7,000 people diagnosed with Gaucher disease worldwide. But Shire is gearing up to treat additional "hundreds" of patients next year, a spokesperson for the drug maker said, and the company has reshuffled some manufacturing in order to produce more velaglucerase.
The capacity constraints have tempered some analyst views on the upside for Shire. Lazard Capital Markets analyst William Tanner downgraded Shire from a 'buy' to 'hold' on Nov. 16, citing the high valuation for an opportunity that "likely cannot be fully seized."
"We believe the stock's recent appreciation, especially in response to [the] announcement of further manufacturing problems for Genzyme, positions the stock for little upside on a fundamental basis," Tanner said in the accompanying research report.
In November alone, Shire's stock price has jumped more than 12 percent, with some of the gains fueled by news Nov. 13 that lots of several of Genzyme's key drugs,including Cerezyme and Fabrazyme (which also competes with Shire's Replagel for Fabry disease outside the U.S.),had been found to contain particles of stainless steel and/or non-latex rubber from vial stoppers.
Genzyme will need to close its Allston, Mass. manufacturing facility for a brief period of time as a result. That news comes after the facility was shut down over the summer for six weeks due to a different bioreactor contamination ('The Pink Sheet,' Sept. 7, 2009).
-Jessica Merrill (j.merrill@elsevier.com)
This article is reprinted from "The Pink Sheet" DAILY –Nov 24, 2009
Click here to start your 30-day, risk-free trial of "The Pink Sheet" DAILY – Immediate business intelligence from the company and product level up.
