Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study.

What was studied

This late-stage extension study looked at pegunigalsidase alfa in adults with Fabry disease. The study is called BRIGHT F51. The drug is an enzyme replacement therapy. Other such therapies need infusions every 2 weeks. This drug lasts longer in the body. It was given every 4 weeks at 2 mg/kg. People had used other enzyme therapies for 3 years or more. This interim report covers 3 to 5 years of treatment.

What they found

The median yearly eGFR slope was -2.2, a measure of kidney function over time. It was -2.6 in people with antibodies to the drug. It was -1.8 in those without. Of 477 adverse events during treatment, 51 were judged to be drug-related. These occurred in 13 patients. All were mild or moderate. Some patients had mild or moderate infusion reactions. One patient developed short-lived antibodies to the drug. In females, lyso-Gb3 levels stayed low and stable.

What it means for patients

The authors say long-term treatment was well tolerated. They say disease stayed stable, especially in females and antibody-negative males. They say clinical outcomes should be closely watched with dosing every 4 weeks.

Why it is not certain yet

More data are needed for antibody-positive males. This is an interim analysis. The final results will further test the dosing plan. The abstract gives no comparison group. The study was open-label, so everyone knew the treatment.

Source

Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study.

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