Pompe Disease: FDA Approves Lumizyme

The Food and Drug Administration has OK'd  the first U.S. treatment specifically for late-onset Pompe disease

Article Highlights:
  • Lumizyme is a laboratory-developed enzyme used to replace the acid maltase enzyme deficient in people with Pompe disease.
  • The drug is the first treatment approved in the United States specifically to treat patients with late-onset Pompe disease.
  • The FDA approval comes more than three weeks ahead of the expected decision date of June 17.
  • Severely affected adults who have been receiving treatment through a temporary access program, and those adults who have been waiting for treatment, should contact their physician to discuss beginning treatment through commercial channels.
by Amy Madsen on May 25, 2010 - 12:34pm

The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.

The decision was reported via press release by the drug's manufacturer, Genzyme of Cambridge, Mass., and comes three weeks ahead of the scheduled decision date of June 17, 2010. (For more on Lumizyme's development, see Lumizyme on the Horizon.)

Lumizyme (alglucosidase alfa) is a laboratory-developed enzyme used to replace the acid maltase enzyme deficient in people with Pompe disease ages 8 and older.

Genzyme received FDA approval in 2006 to market Myozyme, the first-ever U.S. treatment for infants and children with Pompe disease. Due to limited production capacity, Myozyme has been reserved for children 17 and younger.

MDA-supported basic research played a role in the development of these enzyme-replacement drugs.

Meaning for people with Pompe disease

Severely affected adults who have been receiving alglucosidase alfa through a program known as the Alglucosidase Alfa Temporary Access Program (ATAP) will continue to receive therapy as they transition to the commercial supply.

Those who have been waiting for treatment should contact their physician about getting access to the new medication.

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