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: Argentina, Australia, Belgium, Brazil, Canada, Chile, China, Colombia, Estonia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Korea, Latvia, Lebanon, Mexico, Netherlands, Poland, Qatar, Russia, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
Active Clinical Trial Count: 13
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 |
Unknown Status |
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 |