Drug

Cofirasersen

As of the edition of 20 October 2026, Cofirasersen is an oligonucleotide: stopped in 2021; acts on SCNN1A.

Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.

The drug

Status
Stopped, 2021
Kind
Oligonucleotide
Registered trials
2, 1 stopped early
Disease areas
Lungs and airways; Digestive system; Infections; Inherited and neonatal

US protection

No US approval on record.

With no US approval on record there are no listed US patents or exclusivities.

This is the US only.

Companies that need a new molecule because of this drug

1 company on route A because of this drug.

Companies on route A for this drug

In the product

1 company is on route A because of Cofirasersen.

Target

GeneProteinUniProt
SCNN1AEpithelial sodium channel subunit alphaP37088

Registered trials

PhaseTrialsCompletedActiveStopped early
Phase 11100
Phase 21001
All trials2

Who ran its trials

CompanyRoleYears
Akcea TherapeuticsLead sponsor of trials2018 to now