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Apitegromab

Not yet reviewed: Listed from FDA or ClinicalTrials.gov records; an editor has not checked it yetListed from FDA or ClinicalTrials.gov records; an editor has not checked it yet.

Also known as Isembyld, Apitegromab-mstn

Scholar Rock Holding Corp SRRK · Anti-myostatin; Atrophy; Muscular Atrophy; Muscular Atrophy, Spinal; Neuromuscular Diseases

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Key figures

  • Next catalyst

    None scheduled

    Only dates with a source are listed

  • Phase

    Approved

    Not recorded

  • Linked trials

    2

    From ClinicalTrials.gov

  • Latest FDA action

    Approved

    BLA761463,

Recalls, shortages, patents and EU status at a glance

Key information

Sponsor
Scholar Rock Holding Corp SRRK Source: Company record for Scholar Rock Holding Corp (opens in a new tab)
Indications
Anti-myostatin; Atrophy; Muscular Atrophy; Muscular Atrophy, Spinal; Neuromuscular Diseases Source: Conditions from ClinicalTrials.gov (opens in a new tab)
Application
BLA 761463 · Priority review Source: Drugs@FDA via openFDA (opens in a new tab) · Action letter for BLA 761463 (opens in a new tab)
Designations
None recorded
Next catalyst
None scheduled

Development timeline

  1. Preclinical Completed
  2. Phase 1 Completed
  3. Phase 2 Completed
  4. Phase 3 CompletedFirst trial start 14 Apr 20222 linked trials
  5. Filed Completed
  6. Approved Current stageFirst approval 11 Sep 2026

Overview

Mechanism
Not recorded
Route
Not recorded
Modality
Not recorded
Specialties
Not recorded

Overview text is written by HealthTrade editors.

Key dates

  1. ApitegromabPDUFAOccurred

    · PDUFA date · Scholar Rock Holding Corp SRRK

    Scholar Rock Holding Corp: PDUFA date in EX-99.1 (8-K) filed 14 May 2025

    EX-99.1 2 srrk-20250514xex99d1.htm EX-99.1 Exhibit 99.1 Scholar Rock Reports First Quarter 2025 Financial Results and Highlights Business Progress • Apitegromab BLA accepted under priority review for patients with Spinal Muscular Atrophy (SMA) and FDA’s PDUFA date set for September 22, 2025; commercial readiness progresses • European Medicines Agency validated Marketing Authorisation Application (MAA) for apitegromab for patients with SMA • Topline results from Phase 2 EMBRAZE proof-of-concept trial in adult patients with obesity on track for June 2025 • Cash, cash equivalents and marketable securities of $364.4 million as of March 31, 2025; expected to support commercial and development programs into 2027 • Management to host update call today at 8:15 a.m.

    Source: 0001558370-25-007650 (opens in a new tab)

    406days ago

FDA designations

No FDA designations are on record for Apitegromab. Editors add each one with its source.

Filed application

No filed application is on record for Apitegromab. Approvals are listed under FDA actions.

FDA advisory committees

No FDA advisory committee meetings are on record for Apitegromab.

Clinical trials

Trials linked to Apitegromab
TrialTitlePhaseStatusEnrollmentPrimary completionResults posted
NCT05626855ClinicalTrials.gov record for NCT05626855 (opens in a new tab)ONYXLong-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of ApitegromabPhase 3Active, not recruiting238 (actual)1 Nov 2026 (estimated) No
NCT05156320ClinicalTrials.gov record for NCT05156320 (opens in a new tab)SAPPHIREEfficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or RisdiplamPhase 3Completed188 (actual)18 Dec 2024 (actual) No

Publications listing SAPPHIRE (1), newest first

  1. Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial.

    The Lancet. Neurology · 2025 Sep

    PubMed 40818473 (opens in a new tab)DOI 10.1016/S1474-4422(25)00225-X (opens in a new tab)

Citations from PubMed, U.S. National Library of Medicine; abstracts are not stored. A publication lists this study’s NCT number and may be a review that cites many studies.

Source: ClinicalTrials.gov, U.S. National Library of Medicine. Posted results are shown as ClinicalTrials.gov prints them; results from company and journal sources are entered by editors and listed separately. Full result tables are on ClinicalTrials.gov.

Trial quality

About CT-VCA

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FDA actions

Original approvals and new-indication supplements for Apitegromab, newest first
ApplicationActionDateOutcomeReviewSource
BLA761463Action letter for BLA761463 (opens in a new tab) Original 1 Type 1 - New Molecular EntityApprovedpriorityDrugs@FDA via openFDA (opens in a new tab)

Data provided by the U.S. Food and Drug Administration.

FDA recalls load here.

FDA drug shortages load here.

Orange Book patents load here.

FAERS adverse event counts load here.

Papers and preprints load here.

Community discussion loads here.

EU status (EMA)

Application withdrawnOrphan medicine

Isembyld (EMEA/H/C/005909)

Authorised
Not given
Therapeutic area
Muscular Atrophy, Spinal
Marketing authorisation holder
Scholar Rock Netherlands B.V.
Active substance (INN)
apitegromab
ATC code
M09AX

Matched by active substance: EMA's INN or active substance equals this drug's name.

EMA page for Isembyld (opens in a new tab)EMA medicine data (opens in a new tab)

Source: European Medicines Agency (EMA), medicines data download. Licence: EMA re-use with acknowledgement. EMA medicines report 2026-10-26.

Related news

No recent headlines mention this drug.

Sources

Record updated .

Dates come from company and FDA disclosures and can change. Nothing on this page is investment advice.