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SAGE-547

Supernus Pharmaceuticals, Inc. · Phase 3 active Small molecule

SAGE-547 is an intravenous formulation of an antisense oligonucleotide that targets the GABA_A receptor alpha 1 subunit.

SAGE-547 is an intravenous formulation of an antisense oligonucleotide that targets the GABA_A receptor alpha 1 subunit. Used for Status epilepticus.

At a glance

Generic nameSAGE-547
SponsorSupernus Pharmaceuticals, Inc.
Drug classantisense oligonucleotide
TargetGABA_A receptor alpha 1 subunit
ModalitySmall molecule
Therapeutic areaNeurology
PhasePhase 3

Mechanism of action

By binding to the GABA_A receptor alpha 1 subunit, SAGE-547 aims to increase the expression of the alpha 1 subunit, which can help to reduce seizure activity. This is particularly relevant for the treatment of status epilepticus, a severe and life-threatening form of epilepsy. The antisense oligonucleotide mechanism allows for a targeted and specific approach to modulating the GABA_A receptor, which can help to reduce the risk of adverse effects.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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