The U.S. Food and Drug Administration has approved Nexviazyme (ingredient: avalglucosidase alfa-ngpt) for intravenous infusion to treat patients aged one year or above with late-onset Pompe disease (LOPD).

The U.S. Food and Drug Administration has approved Nexviazyme (ingredient: avalglucosidase alfa-ngpt) for treating patients above one and older with late-onset Pompe disease (LOPD).
The U.S. Food and Drug Administration has approved Nexviazyme (ingredient: avalglucosidase alfa-ngpt) for treating patients above one and older with late-onset Pompe disease (LOPD).

LOPD patients have mutated alpha-glucosidase (GAA) gene that reduces or eliminates essential enzymes and causes excessive glycogen accumulation in skeletal and heart muscles, leading to premature death from respiratory or heart failure. This is because healthy people break down glycogen to release glucose into the bloodstream as a fuel for the cells.

“Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, deputy director of the Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research.

The recent approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease. The FDA will continue to work with stakeholders to advance the development of additional new, effective, and safe therapies for rare diseases, including Pompe disease, she added.

Nexviazyme, an enzyme replacement therapy (ERT), is a medication that reduces glycogen accumulation. The drug's effectiveness for Pompe disease was demonstrated in a study of 100 patients comparing Nexviazyme and another authorized ERT for Pompe disease. Patients who received Nexviazyme showed improvement in lung function compared to the control group, the FDA said.

The most common side effects were headache, fatigue, diarrhea, nausea, joint pain, dizziness, muscle pain, itching, vomiting, difficulty breathing, skin redness, and prickling sensation. Severe adverse reactions included hypersensitivity responses such as anaphylaxis and infusion-associated reactions, including respiratory distress, chills, and abnormally elevated body temperature.

Nexviazyme was given the FDA fast-track and orphan drug status to assist and encourage the development of drugs for rare diseases. The agency granted the approval of Nexviazyme to Genzyme Corp.

The company also plans to apply for a regulatory nod to the Ministry of Food and Drug Safety.

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