The Food and Drug Administration has approved a new
drug to treat Pompe disease, a rare and debilitating inherited condition that can lead to death.
Myozyme is the first treatment ever approved for the progressive muscle disorder, which affects only a few thousand people worldwide. The Cambridge, Mass., company has built its business on the treatment of so-called orphan diseases -- a term that's applied to rare illnesses.
"This is a special day for people across the Pompe community and at Genzyme, who have worked together for many years and overcome enormous challenges so that patients with this devastating disease now have a chance," Henri Termeer, Genzyme's chairman and chief executive, said Friday.
People with Pompe disease experience muscle weakness and difficulty breathing, but the severity varies widely. The illness can be fatal, because it eventually leads to respiratory failure. Older people who have it might live for years, but infants can die before the age of 1. At any given time, an estimated 5,000 to 10,000 people globally are living with Pompe.
While Myozyme was shown to improve survival without a ventilator in patients with infantile-onset Pompe, use of the drug in people with other forms of the disease hasn't been studied enough to assure its safety and effectiveness, according to the drug's label.
The labeling also includes a boxed warning indicating that possible life-threatening reactions, like anaphylactic shock, could occur during Myozyme infusions. The warning states that "because of the potential for severe infusion reactions, appropriate medical support measures should be available when Myozyme is administered."
Of the 280 patients who've received Myozyme so far, eight patients, or 3%, have experienced severe or significant hypersensitivity reactions, the company said.
Shares of Genzyme closed down 3 cents at $61.16.