Shire, the British drugmaker with a big presence in Massachusetts, has seen its fortunes rise during this year of manufacturing woes over at Cambridge, MA-based Genzyme. Shire (LON: SHP) doesn’t yet have regulatory approval to market two treatments for genetic diseases that will rival Genzyme, but U.S. regulators have allowed the company to go ahead anyway and start supplying its therapies to patients who are in need, and can’t get access to Genzyme’s drugs.
Sylvie Gregoire, president of Shire Human Genetic Therapies, talked to me this week from her office in Lexington, MA about how her firm has made inroads in the U.S. markets for Fabry and Gaucher’s diseases. Genzyme (NASDAQ: GENZ) has dominated these two markets for years with its Fabry drug, agalsidase beta (Fabrazyme), and its Gaucher’s treatment, imiglucerase (Cerezyme). Together, those two drugs generated $1.7 billion in revenue last year, more than one-third of the company’s total revenue.
But both those drugs have been in short supply since June, when Genzyme revealed that viral contamination was found at its Allston plant in Boston, where both those treatments are made. Shire is hoping to gain FDA approval for its Fabry and Gaucher’s treatments—agalsidase alfa (Replagal) and velaglucerase alfa, respectively—next year. Shire’s timeline for full approval of has been sped up by several months because of the manufacturing trouble over at Genzyme, Gregoire says.
By September, three months after the viral contaminant struck Genzyme, regulators gave Shire permission to give patients its Gaucher’s treatment to address the void in supply for Genzyme’s treatment, which is the market-leading therapy for the disease in the U.S. A similar program to supply Fabry patients with Shire’s drug for that disease began last month. Gregoire would not disclose how many Fabry and Gaucher’s patients are receiving her firm’s therapies, but she did acknowledge that having people already taking the drugs could give her company a head start in marketing the products if the FDA approves them.
“We haven’t announced what percentage of the market we think we will have,” she said, “but whatever market share we are anticipating we expect to gain it more rapidly” because of the current supply shortages. The FDA has granted Shire an expedited approval process for its Gaucher’s treatment. The agency has a deadline of … Next Page »