On May 24, 2010 the Food and Drug Administration (FDA) approved Lumizyme for treatment of “patients 8 years and older with late (non-infantile) onset Pompe disease who do not have evidence of cardiac hypertrophy.”
This is great news for the Pompe community in the USA, the therapy that has been available to the rest of the world (known as “Myozyme” and produced in Belgium at 4000 litre capacity) is now also commercially available to patients in the USA, although the name is changed to “Lumizyme” to avoid confusion with Myozyme produced in the US at 160 litre capacity. Myozyme produced at the lower capacity will be preserved for patients in the US under 8 years who have heart involvement (cardiac hypertrophy).
This will provide wider access to the therapy and means that Genzyme can be reimbursed for the same drug that has, until now, been given freely through the ATAP program.
Please refer to the US Patient Support Group for more information on how the transition to a commercial therapy will affect patients in the USA: www.amda-pompe.org.