"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval
Update (Aug. 4, 2014): The U.S. Food and Drug Administration (FDA) has expanded the approval of Lumizyme so that it can now be used to treat Pompe disease patients of all ages, including children younger than age 8. See FDA Expands Approval of Drug to Treat Pompe Disease to Patients of All Ages; Removes Risk Mitigation Strategy Requirements, Aug. 1, 2014.
Monique Griffin of Orlando, Fla., was one of the first in the nation to receive the commercially available treatment Lumizyme for acid maltase deficiency (AMD, or Pompe disease).
"It was really exciting," Griffin, 35, said of her infusion. Although she’s been receiving treatments under a temporary-access program since March, this was her first treatment since Lumizyme received U.S. Food and Drug Administration (FDA) approval in late May. FDA approval means the drug can be prescribed by physicians and covered by insurance.
"In a way, it was like starting all over again,” said Griffin, who received the infusion at Shands Medical Plaza at the University of Florida (UF) in Gainsville.
Lumizyme is a laboratory-developed enzyme manufactured by Genzyme Corp., of Cambridge, Mass. It replaces the acid maltase enzyme deficient in people with Pompe disease, and is the first treatment approved in the United States specifically for people ages 8 and older with the late-onset form of the disease.
The FDA granted approval for U.S. sales of the drug May 25, 2010. (See Pompe Disease: Genzyme Receives FDA Approval for Lumizyme.)
Lumizyme treatments are administered every two weeks via an approximately four-hour infusion into the arm or hand, followed by a one-hour observation period.
MDA-supported basic research played a role in the development of both Lumizyme and Myozyme, Genzyme's enzyme replacement drug for infants and very young children. (For more, see Lumizyme on the Horizon.)
Making the switch
Griffin learned she has Pompe disease in January 2010. Since March, she has been receiving enzyme infusions through Genzyme's Alglucosidase Alfa Temporary Access Program (ATAP), which the company set up in order to provide treatment for adults who are severely affected by the disease during the time Genzyme was waiting for FDA approval.
|Monique Griffin looks at her medication while receiving a treatment at the University of Florida infusion center at Shands Medical Plaza in Gainesville, Fla.|
A flurry of activity preceded last week's event, which Griffin noted, "almost didn't happen."
"We all had to work around the clock for the week leading up to today in order to put everything in place," Griffin wrote in her blog, M.E.G.'s Confessional – My Mystery Disease is Pompe.
"Phone calls, e-mails, faxes were going non-stop between UF, Genzyme, myself, my insurance company, the pharmaceutical distributor, and the wholesaler. I didn't receive the final OK that today's infusion was going to happen until 8:30 p.m. last night."
Even though she's only been receiving treatments with Lumizyme for a short time, Griffin said she's already noticed "little things," such as being able to walk longer distances and finding it easier to get up from a seated position. "I know it's a slow process," she said, "but I'm convinced Lumizyme has stopped the progression of my disease and had a positive effect."
Griffin is happy that all those in need can now receive Lumizyme. "Now it's available for all adults," she said, "and it's literally saving lives."
Transition to commerical supply
Severely affected adults who have been receiving alglucosidase alfa through 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.