Tofersen

all-P-ambo-2′-O-(2-Methoxyethyl)-5-methyl-P-thiocytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)adenylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioguanylyl-(3’→5′)-2′-O-(2-methoxyethyl)guanylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-2′-deoxy-5-methyl-P-thiocytidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-5-methyl-P-thiocytidylyl-(3’→5′)-P-thiothymidylyl-(3’→5′)-2′-deoxy-P-thioadenylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methylcytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)-P-thioadenylyl-(3’→5′)-2′-O-(2-methoxyethyl)guanylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’→5′)-2′-O-(2-methoxyethyl)-5-methyluridine

C₂₃₀H₃₁₇N₇₂O₁₂₃P₁₉S₁₅  : 7127.86
[2088232-70-4]

Tofersen

CAS 2088232-70-4

FDA APPROVED 4/25/2023, Qalsody

• BIIB 067
• BIIB067
• Formula
  C₂₃₀H₃₁₇N₇₂O₁₂₃P₁₉S₁₅
  Molar mass
  7127.85 g·mol⁻¹

• Antisense Oligonucleotide Inhibitor Of The Expression Of Superoxide Dismutase 1 Gene
• DNA, D((2′-O-(2-METHOXYETHYL))M5RC-SP-(2′-O-(2-METHOXYETHYL))RA-(2′-O-(2-METHOXYETHYL))RG-SP-(2′-O-(2-METHOXYETHYL))RG-(2′-O-(2-METHOXYETHYL))RA-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-T-SP-T-SP-M5C-SP-T-SP-A-SP-(2′-O-(2-METHOXYETHYL))M5RC-(2′-O-(2-METHOXYETHYL))R
• IONIS SOD1Rx

To treat amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation
Drug Trials Snapshot

A nucleic acid-based drug indicated for the treatment of a specific type of amyotrophic lateral sclerosis.

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS).^[3] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. It is administered as an intrathecal injection.^[3]

The most common side effects include fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).^[3]

Tofersen was approved for medical use in the United States in April 2023,^[3][6] and in the European Union in May 2024.^[4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[7]

References

1. ^ “Register of Innovative Drugs”. Health Canada. 3 November 2006. Retrieved 17 April 2025.
2. ^ “Qalsody- tofersen injection”. DailyMed. 25 April 2023. Archived from the original on 8 May 2023. Retrieved 10 June 2023.
3. ^ Jump up to:^{a b c d e f g h i j k l} “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). U.S. Food and Drug Administration (FDA). 25 April 2023. Archived from the original on 25 April 2023. Retrieved 25 April 2023.  This article incorporates text from this source, which is in the public domain.
4. ^ Jump up to:^a b c d “Qalsody EPAR”. European Medicines Agency (EMA). 22 February 2024. Retrieved 24 February 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
5. ^ Jump up to:^a b “Qalsody PI”. Union Register of medicinal products. 3 June 2024. Retrieved 7 September 2024.
6. ^ “FDA Grants Accelerated Approval for Qalsody (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS” (Press release). Biogen. 25 April 2023. Archived from the original on 25 April 2023. Retrieved 25 April 2023 – via GlobeNewswire.
7. ^ Jump up to:^a b New Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024.
8. ^ Liu A (1 May 2019). “Biogen’s antisense ALS drug shows promise in early clinical trial”. FierceBiotech. Archived from the original on 2 February 2023. Retrieved 25 April 2023.
9. ^ Langreth R (22 March 2023). “Biogen’s ALS Drug Gets Partial Backing From FDA Panel”. Bloomberg News. Retrieved 25 April 2023.
10. ^ “FDA approves drug which helps to slow progression of rare form of MND”. http://www.sheffield.ac.uk. 28 April 2023. Retrieved 16 May 2024.
11. ^ Berdyński M, Miszta P, Safranow K, Andersen PM, Morita M, Filipek S, et al. (January 2022). “SOD1 mutations associated with amyotrophic lateral sclerosis analysis of variant severity”. Scientific Reports. 12 (1): 103. Bibcode:2022NatSR..12..103B. doi:10.1038/s41598-021-03891-8. PMC 8742055. PMID 34996976.
12. ^ Constantino A (25 April 2023). “FDA grants accelerated approval for Biogen ALS drug that treats rare form of the disease”. CNBC. Archived from the original on 25 April 2023. Retrieved 25 April 2023.
13. ^ Constantino A (22 March 2023). “FDA advisors vote against effectiveness of Biogen’s ALS drug for rare and aggressive form of the disease”. CNBC. Archived from the original on 10 April 2023. Retrieved 25 April 2023.
14. ^ Robins R (25 April 2023). “F.D.A. Approves Drug for Rare Form of A.L.S.” The New York Times. Archived from the original on 25 April 2023. Retrieved 25 April 2023.
15. ^ “New treatment for rare motor neuron disease recommended for approval”. European Medicines Agency (EMA) (Press release). 23 February 2024. Retrieved 24 February 2024.

////////////tofersen, Qalsody, FDA 2023, APPROVALS 2023, EU 2024, EMA 2024, BIIB 067, BIIB067, IONIS SOD1Rx