Asset profile

Nusinersen · Spinraza

RNA / oligonucleotide

Approved / Phase 4Mechanism not established
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Approvedregulatory verdict

Evidence outlook

Approved: Spinal Muscular Atrophy

Regulatory approval supersedes design-level evidence grading; the trials behind approval are preserved below as the drug's evidence history.

track record: Reliable signal

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Competitive landscape

2 competitors in this universe: 0 direct rivals (same mechanism and indication), 0 same-class, 2 same-indication.

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DrugStageModalityMechanismEvidence signalReported efficacygraded featured trials
Nusinersen (Spinraza): this drugApprovedRNA / oligonucleotide—
ApitegromabPhase 3Monoclonal antibodyActivin/myostatin type II receptor
Taldefgrobep AlfaPhase 3Antibody-derived scaffoldActivin/myostatin type II receptor
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Research adds each competitor's evidence grade and sourced trial results, aligned per race, so you can see who actually has the data.

Upcoming events

(3 total)

Estimated primary-completion dates from ClinicalTrials.gov. These are registry estimates, never confirmed readout dates; they can move as trials progress.

2027 (2)
September 2027 (2)
Phase 3Spinal Muscular AtrophyActive not recruiting
Est. primary completion 2027-09-24 · NCT05067790 · CT.gov ↗
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Phase 1Muscular Atrophy, SpinalRecruiting
Est. primary completion 2027-09-30 · NCT06555419 · CT.gov ↗
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2028 (1)
November 2028 (1)
Phase 2Spinal Muscular AtrophyRecruiting
Est. primary completion 2028-11 · NCT07047144 · CT.gov ↗
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All registered studies

Every ClinicalTrials.gov study of this drug's own program, grouped by indication and phase. Research unlocks the studies themselves: titles, statuses, dates and featured-trial cross-links.

Phase 3 (7)
Phase 2 (4)
Phase 1/2 (1)
Phase 1 (5)
Phase 4 (2)
Early phase / not applicable (3)
Appears in other studies (1)