Last Update: Aug 6, 2023
Click to purchase the complete database for corresponding clinical trial identifiers, clinical trial parameters, discontinued programs, notes on Spinraza, Zolgensma, Evrysdia (e.g exclusivity, patents, key clinical data), reported net product sales of Spinraza, Zolgensma and Evrysdi every quarter from Q4 2016 up to Q3 2022 (including additional market data), a highlight of apitegromab, including patents and Ph2 clinical trial data summary.
| Company | Candidate | MOA | Indications | Administration/Dose | Approval |
|---|---|---|---|---|---|
| Roche (Genentech), PTC Therapeutics | Evrysdi (risdiplam) | SMN2 splicing modifier | Treatment of SMA (Type 1, 2, 3) in patients aged 2 mth and older | Oral QD 0.15-5 mg/kg based on age/weight | Aug 2020 (US), Mar 2021 (EU) |
| Biogen | Spinraza (nusinersen) | Antisense oligonucleotide | Treatment of SMA (Type 1, 2, 3) in pediatric and adult patients | Intrathecal injection 12 mg Q2W first 3 doses, 4th dose after 30 days, then maintenance dose Q4M | Dec 2016 (US), Jun 2017 (EU) |
| Novartis | Zolgensma (onasemnogene abeparvovec-xioi) | scAAV9 containing SMN1 gene under CAG promoter | SMA in patients less than 2 years of age | Single-dose IV infusion 1.1E14 vg/kg | May 2019 (US), May 2020 (EU) |
| Company | Candidate | MOA | Indications | Administration/Dose | Phase |
|---|---|---|---|---|---|
| Scholar Rock | Apitegromab (SRK-015) | Anti-myostatin (latent) IgG4 mAb | Type 2/3 SMA on background nusinersen or risdiplam therapy | IV Q4W 10 or 20 mg/kg | III |
| Novartis/Avexis | OAV101 (AVXS-101, Zolgensma) | SMN gene replacement therapy | Type 2 SMA | Single dose intrathecal 1.2E14 vg | III |
| Biohaven Pharmaceuticals | Taldefgrobep alfa (RG6206/BMS-986089) | Anti-myostatin (active) adnectin IgG1-Fc fusion protein | SMA ambulant and non-ambulant, ages 4-21 | SC injection 35mg/50mg weekly (weight-based) | III |
| Roche/Genentech | RO7204239 (GYM329) | Anti-myostatin (latent) IgG1 mAb | SMA age 2-10, ambulant (in combination with Evrysdi), FSHD | SC abdominal Q4W w/ oral once daily risdiplam | II/III |
| NMD Pharma | NMD670 | ClC-1 inhibitor | Potentially SMA, myasthenia gravis | Oral | I/II |
| Epirium Bio | MF-300 | 15-PGDH inhibitor | SMA | Oral | Preclin |
| Ractigen Therapeutics | RAG-06 | RNAa (small activating RNAs) | SMA | - | Preclin |
| Shift Pharmaceuticals | E1V1.11 | Antisense oligonucleotide (PMO blocking E1) | SMA | - | Preclin |
| Biogen (Ionis) | BIIB115 (ION306) | Antisense oligonucleotide | SMA | - | Preclin |
| Reborna Biosciences | TEC-1 | SMN2 splicing modifier | Neuromuscular disease | - | Disc |
| AurimMed Pharma | - | - | SMA | - | Disc |
| Neurotune AG | NT 1654 (Agrin) | Agrin/neurotrypsin system | Potentially SMA | - | R&D |