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Onpattro FDA Approval History

Reviewed by J.Stewart B.Pharm. Last updated on Aug 15, 2018.

FDA Approved: Yes (First approved August 10, 2018)
Brand name: Onpattro
Generic name: patisiran
Dosage form: Injection
Company: Alnylam Pharmaceuticals, Inc.
Treatment for: Hereditary Transthyretin-Mediated Amyloidosis (hATTR)

Onpattro (patisiran) is a small interfering ribonucleic acid (siRNA) therapeutic for the treatment of polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR).

Dosage and Administration

  • Onpattro is administered via intravenous infusion over approximately 80 minutes. For patients weighing less than 100 kg, the recommended dosage is 0.3 mg/kg every 3 weeks and for patients weighing 100 kg or more, the recommended dosage is 30 mg every 3 weeks.
  • All patients should receive premedication (corticosteroid, acetaminophen, and antihistamines) to reduce the risk of infusion-related reactions.

Warnings and Precautions

  • Infusion-related reactions: Monitor for signs and symptoms during infusion. Slow or interrupt the infusion if clinically indicated. Discontinue the infusion if a serious or life-threatening infusion-related reaction occurs.
  • 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 (e.g., night blindness).
  • Pregnancy: Instruct patients that if they are pregnant or plan to become pregnant while taking Onpattro they should inform their healthcare provider. Advise female patients of childbearing potential of the potential risk to the fetus.

Adverse Reactions

The most frequently reported adverse reactions (that occurred in at least 10% of Onpattro-treated patients and at least 3% more frequently than on placebo) were upper respiratory tract infections and infusion-related reactions.

Development Timeline for Onpattro

DateArticle
Aug 10, 2018Approval FDA Approves Onpattro (patisiran) Targeted RNA-based Therapy for Polyneuropathy Caused by hATTR
Mar 28, 2018Alnylam Presents New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the 16th International Symposium on Amyloidosis
Feb  1, 2018Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Dec 12, 2017Alnylam Completes Submission of New Drug Application to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Nov 20, 2017U.S. Food and Drug Administration (FDA) Grants Alnylam Breakthrough Therapy Designation (BTD) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis with Polyneuropathy
Nov 16, 2017Alnylam Initiates Rolling Submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for Patisiran for the Treatment of Hereditary ATTR (hATTR) Amyloidosis

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.