FDA Approvals Roundup: Nexviazyme

RoundupsRoundups
| 11 August 2021 | By Renee Matthews 

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
 
New approval
Nexviazyme cleared for late-onset Pompe disease
Genzyme’s Nexviazyme (avalglucosidase alfa-ngpt, intravenous infusion) has been approved for treating late-onset Pompe disease in patients aged 1 year or older.
 
Pompe disease is a rare genetic disorder caused by an enzyme deficiency that drives glycogen accumulation in skeletal and heart muscles, leading to muscle weakness and premature death.
 
Approval of Nexviazyme, and an enzyme replacement therapy, was based on findings from the randomized, multinational, double-blinded, Phase 3 COMET study in approximately 100 patients from the indicated population randomized to receive Nexviazyme or Lumizyme. At week 49, patients receiving Nexviazyme had a 2.9-point improvement in lung function compared with baseline. The improvement was 2.4 points greater than that in the comparator group and met the measurement of noninferiority (P = .0074), according to a press release from Sanofi Genzyme. Statistical superiority of Nexviazyme over Lumizyme was not achieved (P = .06).
 
The application was granted fast track, priority review, and breakthrough therapy designations. Nexviazyme received an orphan drug designation.
 

 

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Tags: FDA, US

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