Spinal muscular atrophy (SMA) global products/pipeline

Last Update: Aug 1, 2022

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)
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Global pipeline

Company Candidate MoA Indication Phase
Scholar Rock Apitegromab
(SRK-015)2
Anti-myostatin (latent/inactive) mAb Type 2 and Type 3 SMA (non-ambulatory) as add-on to background SMN therapy (nusinersen or risdiplam). IV dose III
Biohaven Pharmaceuticals Taldefgrobep Alfa (RG6206/BMS-986089)3 Anti-myostatin (active) adnectin (Fc-fusion protein) SMA (ambulatory and non-ambulatory) as adjunctive therapy to nusinersen or risdiplam. SC dose. III
Roche/Genentech GYM329 (RO7204239)4 Anti-myostatin (active) mAb SMA age 2-10, ambulatory (in combination with Evrysdi). SC dose. II/III
Biogen BIIB110 (ALG-801) ActRIIA/B ligand trap SMA I
Ractigen Therapeutics RAG-06 RNAa (small activating RNAs) SMA Preclin
Biogen (Ionis) BIIB1159 Antisense oligonucleotide SMA Preclin
Shift Pharmaceuticals E1V1.11 SMN2 antisense oligonucleotide SMA Preclin
Reborna Biosciences TEC-1 SMN2 splicing modifier SMA Preclin
Imago Pharmaceuticals - - SMA Preclin
Discontinued/Failed
Novartis LMI070 (branaplam)5 SMN2 RNA splicing modulator SMA II
Roche Olexisome6 Mitochondrial pore modulator Type 2, 3 SMA II
Cytokinetics/Astellas Reldesemtiv FSTA (fast skeletal muscle troponin activator) SMA II7
Catalyst Pharmaceuticals Amifampridine Potassium channel blocker SMA Type 3 (ambulant) II8
Non SMA indications not listed.
1. Intrathecal formulation in Phase 3 for older patients with SMA
2. Apitegromab: 12-month Phase 2 TOPAZ data reported April 6 2021. 24-month data reported June 2022. SAPPHIRE Phase 3 trial (NCT05156320, n=204 non-ambulatory) initiated February 24, 2022.
3. Taldefgrobep Alfa: licensed from BMS (was a drug for DMD licensed to Roche but failed to show efficacy in SPITFIRE Ph2/3 trial, terminated trial and withdrew drug - June 2019) Biohaven licensed the drug from BMS on Feb 25 2022. RESILIENT Phase 3 trial (NCT05337553, n=180 ambulatory and non-ambulatory) as adjunctive therapy to nusinersen or risdiplam or post onasemnogene abeparvovec-xioi (Zolgensma) initiated July 7 2022.
4. MANATEE Ph 2/3 trial (NCT05115110, n=180 ambulatory SMA) initiated June 2 2022.
5. Discontinued July 2021 due to competitive landscape/approval of competing drugs.
6. Discontinued June 2018.
7. Reldesemtiv: Phase II in SMA complete in 2018. No update since. In Phase 3 for ALS.
8. Phase 2 trial (NCT03781479) completed 2021. No further development. Drug is approved for LEMS.
9. Potential for extended dosing intervals to Spinraza. Biogen acquired from Ionis in Jan 2022.

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