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The enzyme replacement therapy treats Pompe disease, a rare genetic disorder that affects children.
Sanofi has selected Orsini Specialty Pharmacy as a limited distribution partner for Nexviazyme (avalglucosidase alfa-ngpt). Nexviazyme is used for treating patients one year of age and older with late-onset Pompe disease.
The FDA approved the therapy in August 2021 based on phase 3 data demonstrating improvements in key disease burden measures and its safety profile. Nexviazyme is an enzyme replacement therapy designed to specifically target the mannose-6-phosphate (M6P) receptor, the key pathway for cellular uptake of enzyme replacement therapy in Pompe disease. Nexviazyme has been shown in clinical trials to provide patients with improvements in respiratory function and walking distance.
“Orsini's dedicated Pompe Disease Care Team’s number one priority is providing exceptional care and compassionate support to patients and their families,” Mike Fieri, Orsini’s founder and chief executive officer, said in a statement. “We are proud to expand our partnership with Sanofi and add Nexviazyme to the growing number of enzyme replacement therapies we dispense to patients with rare diseases.”
Orsini offers a nationwide nursing network, allowing patients to pursue in-home treatment or receive training on self-administrated drugs. The company also coordinates the delivery of medications and supplies, and manages the care between prescribers, nurses and patients.
Pompe disease is caused by a genetic dysfunction of the lysosomal enzyme acid alpha-glucosidase. Estimates vary, but its incidence is generally placed at about 1 in 40,000 births in the United States, according to the National Organization for Rare Disorders. First symptoms can occur at any age from birth to late adulthood. Earlier onset compared with later onset is usually associated with faster progression and greater disease severity.