FDA approves Genzyme’s Lumizyme
pharmafile | May 27, 2010 | News story | Sales and Marketing | Genzyme, Lumizyme, Myozyme, Pompe disease
US biotech Genzyme is celebrating FDA approval of its late-onset treatment for Pompe disease Lumizyme.
The green light to market Lumizyme (alglucosidase alfa) in the US took Genzyme by surprise by coming ahead of schedule and providing a much-needed boost to the company.
Genzyme chairman and chief executive Henri Termeer said: “We are grateful to the FDA for their efforts to approve Lumizyme ahead of its scheduled PDUFA date. This is an important day for the Pompe community, especially for those patients with late-onset Pompe disease in the United States who are awaiting treatment for this devastating disease.
Genzyme received a hefty $175 million fine from US regulators in April for manufacturing problems that have affected a string of its products including Myozyme.
Until now Myozyme was the only other available treatment for Pompe disease available in the US, but had been in short supply due to Genzyme’s limited manufacturing capacity and was reserved to treat infants and children who generally have a much more aggressive form of the disease.
Lumizyme, which has the same active ingredient as Myozyme, has been approved to treat patients aged 8 years and over with late-onset (non-infantile) Pompe disease.
Julie Beitz, a drug evaluation director at the FDA’s Centre for Drug Evaluation and Research, said: “Pompe disease is a devastating condition without the appropriate treatment. The approval of Lumizyme will provide an important treatment for patients diagnosed later in life with Pompe disease.”
Lumizyme’s approval included provision for a risk evaluation and mitigation strategy that will restrict its availability through the Lumizyme ACE (Alglucosidase Alfa Control and Education) programme to ensure its use by the correct patient group.
Lumizyme will also have to carry a Boxed Warning because of the risk of anaphylaxis, severe allergic reactions, and immune-mediated reactions.
Pompe disease occurs in an estimated 1 in every 40,000 to 300,000 births and its primary symptom is heart and skeletal muscle weakness, progressing to respiratory weakness and death from respiratory failure.
Brett Wells
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