ION356
- Generic Name
- ION356
Ionis Pharmaceuticals Advances Pipeline with Key Regulatory and Clinical Milestones
• Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and Europe for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, and MAA is under review by the EMA.
• Donidalorsen's NDA for HAE has been accepted by the FDA with a PDUFA date of August 21, 2025, positioning it as a potential prophylactic treatment.
• Phase 3 development of ION582 for Angelman syndrome is set to begin in H1 2025, following positive discussions with the FDA.
Ionis Pharmaceuticals Advances Pipeline with Key Regulatory Milestones and Clinical Data
• Ionis Pharmaceuticals reports positive progress across its pipeline, highlighted by the U.S. launch of WAINUA and advancements in regulatory approvals.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, and the MAA is under review by the EMA, potentially addressing unmet needs.
• Donidalorsen's Phase 3 data positions it as a potential first RNA-targeted prophylactic treatment for HAE, with an FDA action date set for August 21, 2025.
• Positive Phase 2 data for ION582 in Angelman syndrome supports initiation of Phase 3 development in H1 2025, marking progress in neurological disease treatment.
Ionis Pharmaceuticals Highlights Pipeline Progress and Financial Results in Q3 2024
• Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and EU for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, marking a potential treatment for this rare disease.
• Donidalorsen's Phase 3 data positions it as a potential RNA-targeted prophylactic treatment for hereditary angioedema, with an FDA action date set for August 21, 2025.
• The company reaffirms its 2024 financial guidance and increases cash guidance to $2.2 billion, reflecting proceeds from an equity offering.
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