tofersen

Key facts

Generic name

tofersen

Brand name(s)

Qalsody (investigational use outside the approved SOD1 indication)

Gene requirement

SOD1

Genetic confirmation

Not required by this record

ALS population

Adults with non-SOD1 ALS in the Emory-listed study

Category

Investigational treatment

Regulatory status

INVESTIGATIONAL

FDA approval date

Not stated in the official study record.

Approval pathway

Not stated

Country / availability

State-level / commercial availability not verified

Route(s)

Intrathecal administration

Administration

Requires administration or supervision by an experienced healthcare professional.

What is it?

A study program evaluating tofersen outside its FDA-approved SOD1-ALS indication.

Indication: Investigational study use in non-SOD1 ALS; not FDA-approved for this population.

Who is it for?

Adults with non-SOD1 ALS in the Emory-listed study

How does it work?

Mechanism: Antisense oligonucleotide targeting SOD1 biology; use outside the approved SOD1 indication is investigational.

Target: SOD1 pathway

How is it administered?

Intrathecal administration

The official record describes administration by or with experienced healthcare professionals.

Available formulations

Not stated in the official study record.

What evidence supported approval?

The linked ClinicalTrials.gov record is the official study record for this investigational program.

What is known about benefit?

Clinical benefit in non-SOD1 ALS is not established.

Important safety information

Investigational risks and eligibility are determined by the study team and protocol.

This page does not provide dosing, prescribing, or individualized medical advice.

Gene connection

No gene-specific page is linked for this treatment.

Related research

Research opportunities are separate from treatment availability. Eligibility, recruitment, and study status must be confirmed in the Research record.

• Tofersen in Non-SOD1 ALS (NCT07294144)

Official Sources