Chemistry:Apitegromab
Apitegromab, sold under the brand name Isembyld, is a fully human IgG4 lambda monoclonal antibody used for the treatment of spinal muscular atrophy.[1] It is a recombinant monoclonal antibody targeting proforms of myostatin.[1]
The most common side effects include upper respiratory tract infections, vomiting, cough, other viral infections, headache, gastroenteritis, and pharyngitis (sore throat).[2]
Apitegromab was approved for to treat Spinal Muscular Atrophy in the United States in September 2026.[2]
Medical uses
Apitegromab is indicated for the treatment of spinal muscular atrophy in people aged two years of age and older who are currently receiving a survival motor neuron 2-targeted treatment.[1]Spinal muscular atrophy is a rare, progressive neuromuscular disease affecting approximately 1 in 10,000 live births and is among the leading genetic causes of infant mortality.[2] It is caused by a faulty survival motor neuron 1 (SMN1) gene that fails to produce a protein essential for motor neuron survival, leading to progressive muscle weakness and wasting.[2]
Adverse effects
The most common adverse reactions include upper respiratory tract infections, vomiting, cough, other viral infections, headache, gastroenteritis, and pharyngitis (sore throat).[2] An increased risk of fractures, including serious fractures, was observed in people treated with apitegromab.[2] Apitegromab may cause fetal harm and may affect reproductive function.[2]
History
The effectiveness and safety of apitegromab were evaluated in a 52-week randomized, double-blind, placebo-controlled trial (NCT05156320).[2] The trial enrolled 188 participants with spinal muscular atrophy between 2 to 21 years of age who were not able to move or walk independently.[2] All participants were already receiving an approved survival motor neuron 2-targeted treatment.[2] Participants were randomly assigned to receive apitegromab 10 mg/kg, apitegromab 20 mg/kg via intravenous infusion, or placebo once every four weeks for approximately one year.[2] The primary analysis was conducted in 156 participants aged 2 to 12 years of age.[2]
Society and culture
Legal status
In August 2026, Scholar Rock Netherlands withdrew its application for a marketing authorization of Isembyld for the treatment of 5q spinal muscular atrophy.[3] The company stated that the manufacturing site used to make the medicine had not demonstrated compliance with EU GMP requirements within the required time limit.[3]
Apitegromab was authorized for medical use in the United States in September 2026.[4][5]
The US Food and Drug Administration (FDA) granted the application for apitegromab fast track, orphan drug, and rare pediatric disease designations.[2] The FDA granted the approval of Isembyld to Scholar Rock.[2]
Names
Apitegromab is the international nonproprietary name.[6]
Apitegromab is sold under the brand name Isembyld.[4]
References
- ↑ 1.0 1.1 1.2 Cite error: Invalid
<ref>tag; no text was provided for refs namedIsembyld FDA label - ↑ 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 2.11 2.12 2.13 "FDA Approves First Therapy to Target Muscle Loss in Spinal Muscular Atrophy". U.S. Food and Drug Administration (FDA). 11 September 2026. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-therapy-target-muscle-loss-spinal-muscular-atrophy.
This article incorporates text from this source, which is in the public domain.
- ↑ 3.0 3.1 "Isembyld EPAR". 21 August 2026. https://www.ema.europa.eu/en/medicines/human/EPAR/isembyld. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ 4.0 4.1 "Scholar Rock Announces FDA Approval of Isembyld (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular Atrophy (SMA)" (Press release). Scholar Rock. 11 September 2026. Retrieved 15 September 2026 – via business Wire.
- ↑ "FDA Approves Isembyld for People Living with SMA" (Press release). Cure SMA. 14 September 2026. Retrieved 15 September 2026 – via PR Newswire.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information 35 (1). 2021.
Further reading
- Barrett, Doreen; Bilic, Sanela; Chyung, Yung; Cote, Shaun M.; Iarrobino, Ryan; Kacena, Katherine; Kalra, Ashish; Long, Kimberly et al. (2021). "A Randomized Phase 1 Safety, Pharmacokinetic and Pharmacodynamic Study of the Novel Myostatin Inhibitor Apitegromab (SRK-015): A Potential Treatment for Spinal Muscular Atrophy". Advances in Therapy 38 (6): 3203–3222. doi:10.1007/s12325-021-01757-z. ISSN 0741-238X. PMID 33963971.
- Crawford, Thomas; Darras, Basil; Day, John; Song, Guochen; Nomikos, George; Place, Amy; Barrett, Doreen; Bilic, Sanela et al. (3 May 2022). "Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial (P15-5.005)". Neurology 98 (18 Supplement). doi:10.1212/WNL.98.18_supplement.1859. ISSN 0028-3878. https://n.neurology.org/content/98/18_Supplement/1859.
- Crawford, Thomas; Place, Amy; Barrett, Doreen; Cote, Shaun; Nomikos, George; Song, Guochen; Bilic, Sanela; Kalra, Ashish et al. (2021). "Relationship of pharmacokinetics and pharmacodynamics to apitegromab efficacy in patients with later-onset spinal muscular atrophy (Types 2 and 3 SMA): Results from the TOPAZ study". Journal of the Neurological Sciences 429. doi:10.1016/j.jns.2021.118388.
- "Apitegromab for lean mass preservation during tirzepatide-induced weight loss: a randomized, double-blind, placebo-controlled phase 2 trial". Nature medicine: 2673–2678. July 2026. doi:10.1038/s41591-026-04440-4. PMID 42260100.
External links
- "Apitegromab ( Code - C174923 )". https://evsexplore.semantics.cancer.gov/evsexplore/concept/ncit/C174923.
- Clinical trial number NCT05156320 for "Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (SAPPHIRE)" at ClinicalTrials.gov
Template:Other drugs for disorders of the musculo-skeletal system
 |  |
