Drug Profile
Patisiran - Alnylam Pharmaceuticals
Alternative Names: ALN-18328; ALN-TTR02; GENZ-438027; ONPATTRO; Onpattro; Patisiran sodium - Alnylam Pharmaceuticals; patisiran-LNP; SAR-438037Latest Information Update: 05 Nov 2023
Price :
$50
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At a glance
- Originator Alnylam Pharmaceuticals
- Class Small interfering RNA
- Mechanism of Action Prealbumin expression inhibitors; RNA interference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Marketed Amyloid polyneuropathy; Amyloidosis
Most Recent Events
- 09 Oct 2023 Alnylam Therapeutics receives complete response letter from the US FDA for patisiran for Cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis
- 13 Sep 2023 Alnylam Pharmaceuticals announces positive outcome of the US FDA CRDAC meeting for sNDA for patisiran
- 03 Aug 2023 Alnylam Pharmaceuticals completes enrolment in the Expanded Access Program for Amyloidosis in USA (NCT05505838)