🏅 FDA Orphan Designation

AMVUTTRA

VUTRISIRAN

Manufacturer: Alnylam Pharmaceuticals, Inc.

Indicated for:

Hereditary transthyretin-mediated amyloidosisOrphanHereditary ATTR amyloidosisOrphanWild type ATTR amyloidosis

FDA-Approved Indications (3)

Hereditary transthyretin-mediated amyloidosisOrphan Designation

treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart f…

Hereditary ATTR amyloidosisOrphan Designation

Wild type ATTR amyloidosis

Treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart f…

Indications & Usage

1 INDICATIONS AND USAGE AMVUTTRA is a transthyretin-directed small interfering RNA indicated for the treatment of: the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults ( 1.1 ) the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits ( 1.2 ) 1.1 Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. 1.2 Cardiomyopathy of Wild-type or Hereditary Transthyretin-mediated Amyloidosis AMVUTTRA is indicated for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.

Medical disclaimer: This information is for educational purposes only. Always consult a qualified healthcare provider before making treatment decisions. Data sourced from FDA and current as of our last update.