Spinal muscular atrophy (SMA) global products/pipeline

Last Update: April 27, 2021

Approved therapies

Company Product MoA Indication Approval
Biogen Spinraza (nusinersen) Antisense oligonucleotide to promote exon 7 inclusion in SMN2 mRNA Treatment of SMA in pediatric and adult patients - intrathecal dose Dec 2016(US), June 2017 (EU)
Novartis Zolgensma (Onasemnogene abeparvovec-xioi) AAV9 SMN1 gene replacement Pediatric patients less than 2 yrs of age with SMA with bi-allelic mutations in SMN1 gene, regardless of type - single dose IV1 May 2019 (US), May 2020 (EU)
Roche (Genentech)/PTC Evrysdi (risdiplam) SMN2 splicing modifier Treatment of SMA age 2 mths+ - oral dose Aug 2020 (US),
Mar 2021 (EU)

Global pipeline

Company Candidate MoA Indication Phase
Scholar Rock Apitegromab (SRK-015)2 Anti-Myostatin mAb SMA II+
Novartis LMI070 (branaplam) SMN2 RNA splicing modulator SMA II
Biogen BIIB110 (ALG-801) ActRIIA/B ligand trap SMA I
Ractigen Therapeutics RAG-06 RNAa (small activating RNAs) SMA IND-enabling
Shift Pharmaceuticals E1V1.11 SMN2 antisense oligonucleotide SMA Preclin
Reborna Biosciences - RNA-related Neuromuscular disease (SMA) Preclin
Imago Pharmaceuticals - - SMA Preclin
Discontinued/Failed
Roche Olexisome3 Mitochondrial pore modulator Type 2, 3 SMA II
Cytokinetics/Astellas Reldesemtiv FSTA (fast skeletal muscle troponin activator) SMA II4
Non SMA indications not listed.
1. Intrathecal formulation in Phase 3 for older patients with SMA
2. Apitegromab: Data reported April 6 2021. Press release.
3. Discontinued June 2018.
4. Reldesemtiv: Phase II in SMA complete in 2018. No update since. Pursuing in ALS.

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