Drug Profile

Givosiran - Alnylam Pharmaceuticals

Alternative Names: ALN-AS1; GIVLAARI

Latest Information Update: 25 Feb 2026

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At a glance

Originator Alnylam Pharmaceuticals
Class Amides; Amino sugars; Drug conjugates; Pyrrolidines; Small interfering RNA
Mechanism of Action 5-aminolevulinate synthetase inhibitors; RNA interference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Hepatic-porphyria
New Molecular Entity Yes

Highest Development Phases

Marketed Hepatic-porphyria

Most Recent Events

16 Feb 2026 Preregistration for Hepatic-porphyria in South Korea, Saudi Arabia, Serbia (SC)
16 Feb 2026 Registered for Hepatic-porphyria (In adults) in Mexico (SC)
16 Feb 2026 Registered for Hepatic-porphyria (In adults, In adolescents) in Kuwait, United Arab Emirates (SC)

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