Product Description
recently approved antisense oligonucleotide for spinal muscular atrophy (Sourced from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5858681/)
Mechanisms of Action: mRNA Agonist
Novel Mechanism: No
Modality: Nucleic Acid
Route of Administration: Injection
FDA Designation: None *
Approval Status: Approved
Approved Countries: Argentina | Australia | Austria | Belgium | Bosnia | Brazil | Canada | Chile | Colombia | Croatia | Cyprus | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Malaysia | Netherlands | New Zealand | Norway | Poland | Portugal | Romania | Russia | Saudi Arabia | Serbia | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | Ukraine | United Arab Emirates | United Kingdom | United States | Uruguay
Approved Indications: Muscular Atrophy, Spinal | Muscular Atrophy
Known Adverse Events: Constipation
Company: Biogen
Company Location: CAMBRIDGE MA 02142
Company CEO: Michel Vounatsos
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Belgium, Brazil, Canada, Chile, China, Colombia, Estonia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Korea, Latvia, Lebanon, Mexico, Netherlands, Poland, Russia, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
Active Clinical Trial Count: 12
Highest Development Phases
Phase 3: Muscular Atrophy, Spinal
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| jRCT2051230096 | P3 |
Not yet recruiting |
Muscular Atrophy, Spinal |
2030-07-31 |
|
| 232SM303 | P3 |
Not yet recruiting |
Muscular Atrophy, Spinal |
2027-07-22 |
|
| ASCEND | P3 |
Recruiting |
Muscular Atrophy, Spinal |
2027-06-14 |
95% |
| jRCT2031220035 | P3 |
Recruiting |
Muscular Atrophy, Spinal |
2026-11-30 |