- SOD1-ALS affects astir 2% of radical surviving with ALS worldwide1
- If approved, tofersen would beryllium the world's archetypal attraction to people a familial origin of ALS
- EMA acceptance follows FDA's acceptance of tofersen NDA earlier this year
, /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) contiguous announced that the European Medicines Agency (EMA) has accepted the selling authorization exertion (MAA) for reappraisal of tofersen, an investigational medicine for the attraction of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). SOD1-ALS is simply a progressive and uniformly fatal illness that affects less than 1,000 radical crossed Europe.2
The EMA is the 2nd regulatory bureau to judge reappraisal of a selling exertion for tofersen pursuing U.S. Food and Drug Administration (FDA) acceptance earlier this year. The FDA has acceptable a PDUFA day for tofersen of April 25, 2023. There are presently nary treatments targeted for SOD1-ALS.
"EMA acceptance of the tofersen MAA is simply a important improvement for radical and families battling SOD1-ALS," said C. Frank Bennett, Ph.D., enforcement vice president, main technological serviceman and franchise person for neurological programs astatine Ionis. "If approved, tofersen volition beryllium the world's archetypal attraction that targets a familial origin of ALS. Tofersen besides strengthens Ionis' level strategy to people different neurological diseases, including different forms of ALS."
The MAA includes results from the Phase 3 VALOR study, its unfastened statement hold (OLE) study, a Phase 1 survey successful steadfast volunteers and a Phase 1/2 survey evaluating ascending dose levels. Also included are the astir existent 12-month integrated results from VALOR and the OLE survey that were precocious published successful The New England Journal of Medicine.
Biogen announced that it volition support its aboriginal entree programme for tofersen, which is present disposable successful 34 countries. Biogen besides announced that it volition proceed to actively prosecute with different regulators astir the satellite and volition supply updates erstwhile appropriate.
About Tofersen
Tofersen is an antisense medicine being evaluated for the imaginable attraction of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H successful an effort to trim synthesis of SOD1 macromolecule production. In summation to the ongoing unfastened statement hold of VALOR, tofersen is being studied successful the Phase 3 ATLAS survey designed to measure whether tofersen tin hold objective onset erstwhile initiated successful presymptomatic individuals with a SOD1 familial mutation and biomarker grounds of illness activity. Biogen licensed tofersen from Ionis nether a collaborative improvement and licence agreement.
About Amyotrophic Lateral Sclerosis and SOD1-ALS
Amyotrophic lateral sclerosis (ALS) is simply a rare, progressive and fatal neurodegenerative illness that results successful the nonaccomplishment of centrifugal neurons successful the encephalon and the spinal cord that are liable for controlling voluntary musculus movement. People with ALS acquisition musculus weakness and atrophy, causing them to suffer independency arsenic they steadily suffer the quality to move, speak, eat, and yet breathe. Average beingness expectancy for radical with ALS is 3 to 5 years from clip of grounds onset. Patients with immoderate SOD1 mutations person an adjacent shorter beingness expectancy.
Multiple genes person been implicated successful ALS. Genetic investigating helps find if a person's ALS is associated with a familial mutation, adjacent successful individuals without a household past of the disease. Currently, determination are nary genetically targeted attraction options for ALS. Mutations successful the SOD1 cistron are liable for astir 2% of the estimated 168,000 radical who person ALS globally (SOD1-ALS).
About Ionis Pharmaceuticals, Inc.
For much than 30 years, Ionis has been the person successful RNA-targeted therapy, pioneering caller markets and changing standards of attraction with its caller antisense technology. Ionis presently has 3 marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our technological innovation began and continues with the cognition that sick radical beryllium connected us, which fuels our imaginativeness of becoming a leading, afloat integrated biotechnology company.
To larn much astir Ionis sojourn www.ionispharma.com and travel america connected Twitter @ionispharma.
Ionis' Forward-looking Statements
This property merchandise includes forward-looking statements regarding Ionis' concern and the therapeutic and commercialized imaginable of Ionis' technologies, tofersen and different products successful development. Any connection describing Ionis' goals, expectations, fiscal oregon different projections, intentions oregon beliefs is simply a forward-looking connection and should beryllium considered an at-risk statement. Such statements are taxable to definite risks and uncertainties, including those related to the interaction COVID-19 could person connected our business, and including but not constricted to, those related to our commercialized products and the medicines successful our pipeline, and peculiarly those inherent successful the process of discovering, processing and commercializing medicines that are harmless and effectual for usage arsenic quality therapeutics, and successful the endeavor of gathering a concern astir specified medicines. Ionis' forward-looking statements besides impact assumptions that, if they ne'er materialize oregon beryllium correct, could origin its results to disagree materially from those expressed oregon implied by specified forward-looking statements.
Although Ionis' forward-looking statements bespeak the bully religion judgement of its management, these statements are based lone connected facts and factors presently known by Ionis. As a result, you are cautioned not to trust connected these forward-looking statements. These and different risks concerning Ionis' programs are described successful further item successful Ionis' yearly study connected Form 10-K for the twelvemonth ended Dec. 31, 2021, and the astir caller Form 10-Q quarterly filing, which are connected record with the Securities and Exchange Commission. Copies of these and different documents are disposable from the Company.
In this property release, unless the discourse requires otherwise, "Ionis," "Company," "we," "our," and "us" refers to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is simply a trademark of Ionis Pharmaceuticals, Inc.
1 Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Epub 2021 Jul 9.
2 Brown CA, Lally C, Kupelian V, Flanders WD. Estimated Prevalence and Incidence of Amyotrophic Lateral Sclerosis and SOD1 and C9orf72 Genetic Variants. Neuroepidemiology. 2021;55(5):342-353. doi: 10.1159/000516752. Epub 2021 Jul 9.
SOURCE Ionis Pharmaceuticals, Inc.
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