Drugs / Tofersen
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed amyotrophic lateral sclerosis 28 2 milestones | |||||
| Filed | EU (EMA) | — | amyotrophic lateral sclerosis 28 | 2022-12-05 | “Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for review of tofersen” ionis.com ↗ “Biogen Inc. (Nasdaq: BIIB) announced the European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for review of tofersen” biogen.com ↗ |
| Filed | US (FDA) | — | amyotrophic lateral sclerosis 28 | — | “The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval.” biogen.com ↗ “The FDA accepted the tofersen NDA in July 2022 under the accelerated pathway and granted priority review.” biogen.com ↗ |
| Filed amyotrophic lateral sclerosis type 23 1 milestone | |||||
| Filed | US (FDA) | — | amyotrophic lateral sclerosis type 23 | 2022-07-26 | “today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen” biogen.com ↗ |
| Approved Indication not stated 2 milestones | |||||
| Approved | EU (EMA) | Qalsody | — | 2024-05-29 | europa.eu ↗ |
| Approved | US (FDA) | QALSODY | — | 2023-04-25 | fda.gov ↗ |
Trials 7
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03764488 | Dec 2018 → Jun 2021 | — | Biogen | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT07294144 | Dec 2025 → Jan 2027 expected | amyotrophic lateral sclerosis | Washington University School of Medicine | Recruiting | No outcome recorded |
| Phase 33 trials · 1 missed | ||||||
| Phase 3 | NCT04856982 | May 2021 → Aug 2027 expected | familial amyotrophic lateral sclerosis | Biogen | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03070119 | Mar 2017 → Aug 2024 | familial amyotrophic lateral sclerosis | Biogen | Completed | No outcome recorded |
| Phase 3 | NCT02623699 | Jan 2016 → Jul 2021 | amyotrophic lateral sclerosis | Biogen | Completed | Missed primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT07223723 | Dec 2025 → Dec 2027 expected | amyotrophic lateral sclerosis | Biogen | Recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT04972487 | — | amyotrophic lateral sclerosis type 1 | Biogen | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 22
| Date | Issuer | Release |
|---|---|---|
| 2025-12-22 | Biogen | Results The Journal of the American Medical Association (JAMA) Neurology Publishes Long Term Results from the QALSODY Phase 3 VALOR Study and its Open-Label Extension in SOD1-ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) – today announced that The Journal of the American Medical Association (JAMA) Neurology has published final results from the completed Phase 3 VALOR study and its open-label extension (OLE) study evaluating QALSODY ® (tofersen) for the treatment of superoxide dismutase 1 ( SOD1 ) amyotrophic lateral sclerosis (ALS) with over 3.5 years of follow-up. |
| 2024-05-30 | Biogen | Regulatory Biogen Receives European Commission Approval for QALSODY® (tofersen), the First Therapy to Treat a Rare, Genetic Form of ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) announced the European Commission (EC) has granted marketing authorization under exceptional circumstances and maintained orphan designation for QALSODY ® (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 gene ( SOD1 -ALS). |
| 2024-02-23 | Biogen | Regulatory Biogen’s QALSODY® (tofersen), the First Therapy to Treat Rare, Genetic Form of ALS, Received Positive Opinion from CHMP biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending a marketing authorization under exceptional circumstances for QALSODY ® (tofersen) for the treatment of adults with amyotrophic lateral sclerosis (ALS), associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. |
| 2023-04-25 | Ionis Pharmaceuticals | Regulatory FDA approves QALSODY™ (tofersen) as the first treatment targeting a genetic cause of ALS ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that its partner Biogen has received U.S. Food and Drug Administration (FDA) approval of QALSODY™ (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. |
| 2023-04-25 | Biogen | Regulatory 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 biogen.com ↗ |
| 2023-03-22 | Ionis Pharmaceuticals | Regulatory Ionis announced FDA advisory committee voted unanimously for a potential accelerated approval of tofersen for SOD1-ALS ionis.com ↗
Ionis (Nasdaq: IONS) today announced the outcome of the U.S. Food and Drug Administration's (FDA) Peripheral and Central Nervous System Drugs Advisory Committee meeting on tofersen, an investigational antisense medicine for the treatment of people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2023-03-22 | Biogen | Regulatory Biogen Provides Update on FDA Advisory Committee Meeting on Tofersen for SOD1-ALS biogen.com ↗
Additionally, the committee discussed both of these topics and reached consensus that the benefit-risk profile was favorable based on the review of the totality of data for tofersen in people with SOD1-ALS. |
| 2023-01-23 | Biogen | Regulatory Biogen Shares Update on FDA Advisory Committee Meeting for Tofersen biogen.com ↗ |
| 2022-12-05 | Ionis Pharmaceuticals | Regulatory Ionis announces European Medicines Agency accepts Marketing Authorization Application of tofersen to treat rare, genetic form of ALS ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for review of tofersen, an investigational medicine for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2022-12-05 | Biogen | Regulatory European Medicines Agency Accepts Tofersen Marketing Authorization Application to Treat Rare, Genetic Form of ALS biogen.com ↗ |
| 2022-10-17 | Biogen | Regulatory Biogen Announces FDA’s 3-Month Extension of Review Period for the New Drug Application for Tofersen biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has extended the review period of the new drug application (NDA) for tofersen by three months. |
| 2022-09-21 | Biogen | Results The New England Journal of Medicine Publishes Pivotal Tofersen Data that Show Benefits in Rare, Genetic Form of ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that The New England Journal of Medicine (NEJM) has published detailed results from the Phase 3 VALOR study and the combined analysis of VALOR and its open label extension (OLE) study evaluating tofersen for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2022-07-26 | Ionis Pharmaceuticals | Regulatory Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS ionis.com ↗
Ionis Pharmaceuticals (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational antisense medicine for the treatment of superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS). |
| 2022-07-26 | Biogen | Regulatory FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2022-06-03 | Ionis Pharmaceuticals | Results Ionis partner Biogen announces that results from Phase 3 VALOR study and open-label extension of tofersen showed clinical benefit in SOD1-ALS patients ionis.com ↗
The data show that earlier initiation of tofersen, compared to delayed initiation, slowed declines in clinical function, respiratory function, muscle strength and quality of life. |
| 2022-06-03 | Biogen | Results New 12-Month Tofersen Data Presented at ENCALS Meeting Show Clinically Meaningful Benefit in People With SOD1-ALS biogen.com ↗
The data show that earlier initiation of tofersen compared to delayed initiation (six months later in the open-label extension [OLE] study) slowed declines in clinical function, respiratory function, muscle strength, and quality of life. |
| 2021-10-17 | Ionis Pharmaceuticals | Results Ionis' partner Biogen provides update on tofersen Phase 3 VALOR study in SOD1-ALS ionis.com ↗
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) partner Biogen today announced topline results from its placebo-controlled pivotal Phase 3 VALOR study of tofersen (BIIB067), an investigational antisense medicine being evaluated for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2021-10-17 | Biogen | Results Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced topline results from its pivotal Phase 3 VALOR study of tofersen (BIIB067), an investigational antisense drug being evaluated for people with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). |
| 2021-10-14 | Ionis Pharmaceuticals | Results Topline Results from Tofersen Phase 3 Study and its Open Label Extension in SOD1-ALS to be Presented at the American Neurological Association Annual Meeting ionis.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced topline results from its pivotal Phase 3 VALOR study of tofersen (BIIB067) |
| 2021-10-14 | Biogen | Results Topline Results from Tofersen Phase 3 Study and its Open Label Extension in SOD1-ALS to be Presented at the American Neurological Association Annual Meeting biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced topline results from its pivotal Phase 3 VALOR study of tofersen (BIIB067), an investigational antisense oligonucleotide (ASO) drug being evaluated for people with superoxide dismutase 1 ( SOD1 ) amyotrophic lateral sclerosis (ALS), will be presented at the upcoming American Neurological Association (ANA) 2021 virtual meeting, October 17-19, 2021. |
| 2020-07-08 | Biogen | Results The New England Journal of Medicine Publishes Final Results from Phase 1/2 Study of Tofersen for a Genetic Form of ALS biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) today announced that positive results from a Phase 1/2 study of tofersen (BIIB067) for the potential treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS) were published in The New England Journal of Medicine . |
| 2019-05-01 | Biogen | Results Biogen to Present New Interim Data from Its Phase 1/2 Clinical Study of Tofersen (BIIB067) for the Potential Treatment of a Subtype of Familial Amyotrophic Lateral Sclerosis (ALS) biogen.com ↗
Biogen Inc. (Nasdaq: BIIB) announced today interim results of a phase 1/2 study of tofersen, an antisense oligonucleotide (ASO) being studied for the potential treatment of amyotrophic lateral sclerosis (ALS) in adults with a confirmed superoxide dismutase 1 (SOD1) mutation. |
All press releases naming this drug 23 releases
Evidence & citations 8 cited values
Every value below carries the sentence it was read from. 10 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Tofersen | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04972487 ↗ |
| Known as | BIIB-067 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3833346 ↗ |
| Known as | ISIS-SOD1Rx | ChEMBL registry synonym — accepted as the source's own label CHEMBL3833346 ↗ |
| Known as | Qalsody | ChEMBL registry synonym — accepted as the source's own label CHEMBL3833346 ↗ |
| Action | Inhibit | “designed to reduce SOD1 protein synthesis” PMID 41661214 ↗ Feb 2026 |
| Modality | Antisense oligonucleotide | “Tofersen is an antisense oligonucleotide” PMID 32640130 ↗ Jul 2020 |
| Route | Intrathecal | “Intrathecal administration of tofersen is being studied” PMID 32640130 ↗ Jul 2020 |
| Target | SOD1 | “Tofersen, an intrathecal antisense oligonucleotide designed to reduce SOD1 protein synthesis” PMID 41661214 ↗ Feb 2026 |