{
 "query": "vutrisiran",
 "ingredient": "vutrisiran",
 "slug": "vutrisiran",
 "rxcui": "2604578",
 "brands": [
  "Amvuttra"
 ],
 "names": [
  "vutrisiran",
  "vutrisiran sodium"
 ],
 "classes": [],
 "alternatives": [],
 "label": {
  "route_mode": "SUBCUTANEOUS",
  "route_counts": {
   "SUBCUTANEOUS": 1
  },
  "boxed_warning": "",
  "indications_and_usage": "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.",
  "contraindications": "None. None. ( 4 )",
  "warnings_and_cautions": "Reduced serum vitamin A levels and recommended supplementation: Supplement with the recommended daily allowance of vitamin A. Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur. ( 5.1 ) 5.1 Reduced Serum Vitamin A Levels and Recommended Supplementation AMVUTTRA treatment leads to a decrease in serum vitamin A levels [see Adverse Reactions (6.1) and Clinical Pharmacology (12.2) ] . Supplementation at the recommended daily allowance of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the recommended daily allowance of vitamin A should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).",
  "warnings": "",
  "adverse_reactions": "The following clinically significant adverse reactions are discussed in greater detail in other sections of the labeling: Reduced Serum Vitamin A Levels and Recommended Supplementation [see Warnings and Precautions (5.1) ] The most common adverse reactions (\u22655%) were pain in extremity, arthralgia, dyspnea, and vitamin A decreased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alnylam Pharmaceuticals at 1-877-256-9526 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of AMVUTTRA cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Two Phase 3 randomized, multi-center studies evaluated the safety of AMVUTTRA in 448 transthyretin-mediated amyloidosis (ATTR) patients, including 122 patients with hATTR-PN (HELIOS-A) and 326 patients with ATTR-CM (HELIOS-B) [see Clinical Studies (14) ] . In both studies, patients were instructed to take the recommended daily allowance of vitamin A [see Warnings and Precautions (5.1) ] . Polyneuropathy of Hereditary Tr...",
  "drug_abuse_and_dependence": "",
  "controlled_substance": "",
  "recent_major_changes": "Indication and Usage ( 1 ) 3/2025",
  "information_for_patients": "Recommended Vitamin A Supplementation Inform patients that AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Instruct patients to take the recommended daily allowance of vitamin A. Advise patients to contact their healthcare provider if they experience ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness) and refer them to an ophthalmologist if they develop these symptoms [see Warnings and Precautions (5.1) ] . Pregnancy Instruct patients that if they are pregnant or plan to become pregnant while taking AMVUTTRA they should inform their healthcare provider. Inform patients of the potential risk to the fetus, including that AMVUTTRA treatment leads to a decrease in serum vitamin A levels [see Use in Specific Populations (8.1) and Clinical Pharmacology (12.2) ] .",
  "spl_medguide": "",
  "clinical_studies": "14.1 Polyneuropathy of Hereditary Transthyretin-mediated Amyloidosis The efficacy of AMVUTTRA was evaluated in a randomized, open-label clinical trial in adult patients with hATTR-PN (HELIOS-A; NCT03759379). Patients were randomized 3:1 to receive 25 mg of AMVUTTRA subcutaneously once every 3 months (N=122), or 0.3 mg/kg patisiran intravenously every 3 weeks (N=42) as a reference group. Ninety-seven percent of AMVUTTRA-treated patients and 93% of patisiran-treated patients completed at least 9 months of the assigned treatment. Efficacy assessments were based on a comparison of the AMVUTTRA arm of HELIOS-A with an external placebo group in another study (NCT01960348) composed of a comparable population of adult patients with polyneuropathy caused by hATTR amyloidosis. The primary efficacy endpoint was the change from baseline to Month 9 in modified Neuropathy Impairment Score +7 (mNIS+7). The mNIS+7 is an objective assessment of neuropathy and comprises the NIS and Modified +7 composite scores. In the version of the mNIS+7 used in the trial, the NIS objectively measures deficits in cranial nerve function, muscle strength, and reflexes, and the +7 assesses postural blood pressure, qu...",
  "brand_name": "AMVUTTRA",
  "manufacturer": "Alnylam Pharmaceuticals, Inc.",
  "application_number": "NDA215515",
  "pharm_class_epc": [],
  "route": [
   "SUBCUTANEOUS"
  ],
  "effective_time": "20251106",
  "set_id": "8db0facb-81b6-4006-9239-27dc6409c5d3",
  "has_boxed_warning": false,
  "controlled": false,
  "mentions_rems": false
 },
 "faers": {
  "total": 2534,
  "serious": 2127,
  "death": 635,
  "hospitalization": 1013,
  "primary_suspect": 2532,
  "top_reactions": [
   [
    "Death",
    501
   ],
   [
    "Hospitalisation",
    311
   ],
   [
    "Intentional Dose Omission",
    201
   ],
   [
    "Product Dose Omission Issue",
    133
   ],
   [
    "Fall",
    115
   ],
   [
    "Fatigue",
    100
   ],
   [
    "Therapy Interrupted",
    85
   ],
   [
    "Dyspnoea",
    84
   ],
   [
    "Cardiac Failure",
    78
   ],
   [
    "Gait Disturbance",
    75
   ],
   [
    "Asthenia",
    68
   ],
   [
    "Pain In Extremity",
    68
   ],
   [
    "Arthralgia",
    67
   ],
   [
    "Atrial Fibrillation",
    63
   ],
   [
    "Pain",
    60
   ]
  ],
  "by_year": {
   "2019": 1,
   "2021": 1,
   "2022": 23,
   "2023": 367,
   "2024": 516,
   "2025": 868,
   "2026": 758
  },
  "by_year_primary": {
   "2019": 1,
   "2021": 1,
   "2022": 23,
   "2023": 367,
   "2024": 515,
   "2025": 867,
   "2026": 758
  }
 },
 "faers_base": {
  "total": 20692304,
  "serious_share": 0.5742697381596559,
  "death_share": 0.09102355155810585
 },
 "recalls": [],
 "recall_total": 0,
 "drugsfda": {
  "applications": 1,
  "nda": 1,
  "anda": 0,
  "first_approval": "20220613",
  "rems": false,
  "rems_last": "",
  "labeling_supplements": 2,
  "labeling_recent": 2,
  "sponsors": [
   "ALNYLAM PHARMS INC"
  ],
  "unapproved": false,
  "first_approval_fmt": "06/13/2022"
 },
 "trials": {
  "total": 6,
  "completed": 2,
  "terminated": 0,
  "with_results": 3,
  "industry": 5,
  "phase3": 4,
  "results_share": 1.5,
  "terminated_share": 0.0
 },
 "pubmed": {
  "rct": 19,
  "meta": 2,
  "recent_rct": 19,
  "cochrane": []
 },
 "payments": {
  "year": "2024",
  "usd": 1827595.89,
  "records": 6747,
  "by_name": [
   [
    "Amvuttra",
    1827595.89
   ]
  ],
  "names_checked": [
   "vutrisiran",
   "Amvuttra"
  ],
  "source": "bulk-index"
 },
 "partd": {
  "year": "2024",
  "claims": 1192,
  "beneficiaries": 409,
  "spend": 140924646.88,
  "by_brand": [
   {
    "brand": "Amvuttra",
    "claims": 1192,
    "per_claim": 118225.3749,
    "is_generic": false
   }
  ],
  "per_claim": 118225.37489932886
 },
 "mdl": [],
 "dsc": [],
 "settlements": [],
 "prescrire": null,
 "eml": {
  "listed": false,
  "status": "",
  "name": "",
  "matched": null,
  "stewardship": []
 },
 "exposure": null,
 "scores": {
  "safety": {
   "score": 88,
   "letter": "A",
   "why": [
    "Death appears in 2.8x the share of reports seen across all of FAERS (-12)"
   ],
   "weight": 0.35
  },
  "evidence": {
   "score": 35,
   "letter": "F",
   "why": [
    "19 randomized trials and 2 systematic reviews/meta-analyses indexed in PubMed",
    "No Cochrane review found (-5)"
   ],
   "weight": 0.25
  },
  "regulatory": {
   "score": 81,
   "letter": "B",
   "why": [
    "No recalls on record since 2016",
    "2 label changes approved since 2021 (-4)",
    "Label lists recent major changes to warnings or indications (-5)",
    "Approved within the last 5 years: long-term safety is not yet known (-10)"
   ],
   "weight": 0.15
  },
  "conflict": {
   "score": 70,
   "letter": "C",
   "why": [
    "Pharma paid doctors $1,827,596 tied to this drug last year (-30)",
    "Not named in any pending federal MDL"
   ],
   "weight": 0.15
  },
  "options": {
   "score": 40,
   "letter": "F",
   "why": [
    "No generic version approved: brand-only pricing (-30)",
    "No same-class alternatives found: little leverage to switch (-30)"
   ],
   "weight": 0.1
  }
 },
 "total": 66,
 "grade": "C",
 "questions": [
  "The most-reported problems in FDA's adverse event data are death, hospitalisation, intentional dose omission, product dose omission issue. How would we recognize and handle those?",
  "There is no generic. Is there an older generic in the same class that works as well for my situation?",
  "The manufacturer pays physicians heavily to promote this drug. Have you received anything from them?",
  "What result are we expecting, by when, and what is the plan if it does not happen?",
  "If 100 people like me took this drug, how many would actually get better because of it, and how many would be harmed by it? (Doctors call these the number needed to treat and the number needed to harm.)"
 ]
}