Isembyld (apitegromab)

Orphan

Region: 🇪🇺 EU
Active substance: apitegromab
Company: Scholar Rock Netherlands B.V.
Status: Application withdrawn
Therapeutic area: Muscular Atrophy, Spinal
Designations: Orphan
Application: EMEA/H/C/005909
ATC: M09AX
Official record: www.ema.europa.eu

Indication

Treatment of muscular atrophy in patients aged 2 years and older, with 5q spinal muscular atrophy (SMA), who are non-ambulatory and currently receiving a survival motor neuron 2 (SMN2) targeted treatment

Regulatory milestones

Fri 11 Sep 2026🇺🇸 FDA approval

FDA approves Isembyld (apitegromab-mstn), a new medicine

Scholar Rock Inc; BLA761463; New biologic, Priority review; injectable · source

Thu 13 Aug 2026🇪🇺 Application withdrawn (EU)

Isembyld (apitegromab): EU application withdrawn

Scholar Rock Netherlands B.V. · source

Same substance in other markets

🇺🇸 Isembyld Approved · Scholar Rock Inc

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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.