Donidalorsen
- Generic Name
- Donidalorsen
Oncotelic Therapeutics Completes Phase 1 Trial of OT-101/IL-2 Combination for Advanced Solid Tumors
• Oncotelic Therapeutics has successfully completed a Phase 1 trial evaluating OT-101, an antisense therapeutic targeting TGFβ2, in combination with IL-2 for advanced or metastatic solid tumors.
• The combination demonstrated a tolerable safety profile with no unexpected safety signals, positioning the company to advance into further clinical studies exploring synergies with checkpoint inhibitors.
• CEO Dr. Vuong Trieu presented findings at the SWCR 2025 Conference, highlighting TGFβ2's central role in immune suppression and OT-101's ongoing development in pancreatic cancer, gliomas, and combination immunotherapy regimens.
Ionis and Ono Forge $940 Million Deal for Sapablursen in Polycythemia Vera Treatment
• Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront.
• Sapablursen, currently in Phase 2 trials, has received FDA Fast Track and Orphan Drug designations and works by increasing hepcidin production to regulate iron homeostasis in PV patients.
• Ono will gain exclusive global rights for development and commercialization, while Ionis will complete the ongoing IMPRSSION study before transferring responsibilities.
Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments
• Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.
• KalVista Pharmaceuticals' sebetralstat receives Orphan Drug Designation in Japan, marking progress toward the first oral on-demand treatment for HAE attacks.
• The global angioedema pipeline includes 20+ companies developing innovative therapies, with several promising candidates in late-stage development from companies like ADARx, BioMarin, and Astria Therapeutics.
Donidalorsen Shows Sustained Efficacy in Hereditary Angioedema Prophylaxis
• Donidalorsen demonstrates a sustained reduction in HAE attack rates and improved quality of life in patients, according to Phase 3 and Phase 2 clinical program analyses.
• The investigational RNA-targeted medicine is under FDA review, with a PDUFA date set for August 21, 2025, positioning it for potential commercial launch.
• Data from the OASISplus study show that donidalorsen effectively reduces HAE attack rates and improves quality of life in patients switching from other prophylactic treatments.
• Long-term analysis of the Phase 2 open-label extension study reveals that donidalorsen maintains a 96% reduction in HAE attack rates from baseline up to three years.
Major Breakthroughs in HAE Treatment: 2024 Highlights Gene Editing and Novel Therapeutics
2024 marked significant advances in hereditary angioedema (HAE) treatment, with Intellia's gene-editing therapy NTLA-2002 showing a remarkable 95% reduction in monthly attacks. The year also saw important developments in multiple therapeutic candidates, including donidalorsen and garadacimab, while established treatments like Takhzyro demonstrated continued efficacy in adolescent populations.
Ionis' Tryngolza Approved for FCS as Arrowhead's Plozasiran Awaits FDA Review
• The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS).
• Tryngolza, an RNA-targeted therapy, significantly reduces triglyceride levels and the risk of acute pancreatitis in FCS patients when used with a low-fat diet.
• Arrowhead Pharmaceuticals' plozasiran, another RNA interference therapeutic for FCS, has been accepted for FDA review, with a decision expected by November 2025.
• Clinical trials showed Tryngolza achieved a 57% placebo-adjusted mean reduction in triglycerides at 12 months, while plozasiran demonstrated an 80% median reduction.
FDA Accepts New Drug Application for Donidalorsen for Hereditary Angioedema
• The FDA has accepted a new drug application for donidalorsen, an RNA-targeting drug, for prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older.
• Clinical data from Phase 3 OASIS-HAE, OASISplus studies, and an ongoing Phase 2 open-label extension study support the application, demonstrating a 96% reduction in HAE attacks.
• Donidalorsen offers convenient monthly or bi-monthly self-administration via autoinjector and has shown a favorable safety profile with mainly injection-site reactions as adverse events.
• The FDA has set an action date of August 21, 2025, for donidalorsen, which could be Ionis' second independent commercial launch.
FDA Accepts Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' NDA for donidalorsen, a potential prophylactic treatment for hereditary angioedema (HAE) in patients aged 12 and older.
• Donidalorsen, an RNA-targeted medicine, aims to prevent HAE attacks, offering a novel approach to managing this rare genetic condition.
• The FDA has set a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025, for the decision on donidalorsen's approval.
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 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, potentially addressing an unmet need.
• Donidalorsen's NDA for HAE has been accepted by the FDA, with a PDUFA date of August 21, 2025, positioning it as a novel prophylactic treatment.
• Phase 3 results for olezarsen in severe hypertriglyceridemia and pelacarsen in Lp(a)-driven cardiovascular disease are expected next year.
Ionis Pharmaceuticals Highlights Pipeline Progress and Financials in Q3 2024
• Ionis Pharmaceuticals reports strong progress with WAINUA launch for ATTRv-PN, generating $5 million in royalties for Q3 2024, and securing approvals in the UK and a positive CHMP opinion in Europe.
• The company is advancing olezarsen for FCS, with a PDUFA date set for December 19, 2024, and donidalorsen for HAE, with a PDUFA date of August 21, 2025, positioning them for potential launches.
• Positive Phase 2/3 DEVOTE study data for higher dose nusinersen in SMA was presented, with global regulatory applications planned, alongside QALSODY's marketing approval in China for SOD1-ALS.
• Ionis reaffirms its 2024 financial guidance, increasing cash guidance to $2.2 billion, reflecting proceeds from an equity offering to support pipeline advancements and commercial preparations.
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.
Ionis Pharmaceuticals Advances Pipeline with Key Regulatory and Clinical Milestones
• Ionis Pharmaceuticals reports positive progress with WAINUA, achieving commercial success and regulatory approvals for ATTRv-PN treatment.
• Olezarsen's NDA for FCS is under FDA Priority Review with a PDUFA date of December 19, 2024, potentially addressing an unmet need.
• Donidalorsen's NDA for HAE has been accepted by the FDA, with a PDUFA date of August 21, 2025, positioning it as a novel prophylactic treatment.
• The company is advancing multiple Phase 3 programs, including olezarsen for sHTG and ION582 for Angelman syndrome, with key data expected in the coming years.
Cell Therapy and Targeted Therapies Dominate Oncology Advances in Early 2025
• The FDA issued a CRL for Atara Biotherapeutics' tabelecleucel due to third-party manufacturing issues, not efficacy or safety data, delaying potential approval for EBV+ PTLD.
• EsoBiotec dosed the first patient in a trial for ESO-T01, an in vivo BCMA-directed CAR-T therapy for multiple myeloma, aiming for lower costs and simplified administration.
• Obecabtagene autoleucel (obe-cel) gained FDA approval for relapsed/refractory B-cell precursor ALL, offering a less toxic CD19-directed CAR T-cell therapy option.
• Arlocabtagene autoleucel (arlo-cel) shows promise in heavily pretreated relapsed/refractory multiple myeloma, eliciting a 48% complete response rate in phase 1 studies.
FDA Accepts Ionis' Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' NDA for donidalorsen, an RNA-targeted medicine, for prophylactic treatment of hereditary angioedema (HAE).
• The FDA's decision is based on positive Phase 3 OASIS-HAE and OASISplus study results, with a PDUFA action date set for August 21, 2025.
• Donidalorsen aims to reduce prekallikrein production, potentially preventing HAE attacks, with Phase 3 data showing significant attack reduction.
• The drug was well-tolerated in studies, and Ionis is preparing for a potential launch, while Otsuka plans to submit an MAA to the EMA.
FDA Accepts Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' NDA for donidalorsen, an investigational RNA-targeted medicine, for prophylactic treatment of hereditary angioedema (HAE) in patients aged 12 and older.
• Donidalorsen is designed to reduce prekallikrein (PKK) production, interrupting the pathway that leads to HAE attacks, a rare genetic condition causing severe swelling.
• The FDA has set a Prescription Drug User Fee Act (PDUFA) date of August 21, 2025, for donidalorsen, marking a significant step toward potential approval.
• Phase 3 OASIS-HAE study data showed statistically significant improvements in quality of life and disease control with donidalorsen treatment compared to placebo.
FDA Accepts Ionis' Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' NDA for donidalorsen, a potential first-in-class RNA-targeted medicine, for hereditary angioedema (HAE) prophylaxis.
• The FDA's decision is based on positive Phase 3 OASIS-HAE and OASISplus study results, along with ongoing Phase 2 open-label extension study data.
• Donidalorsen targets prekallikrein (PKK) to reduce HAE attack frequency, with a PDUFA date set for August 21, 2025.
• Clinical trials demonstrated a 96% mean reduction in HAE attack rates, with donidalorsen generally well-tolerated among patients.
FDA Accepts Ionis' Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' New Drug Application (NDA) for donidalorsen, a potential first-in-class RNA-targeted medicine.
• Donidalorsen is intended for prophylactic treatment to prevent hereditary angioedema (HAE) attacks in patients aged 12 and older.
• The FDA has set a PDUFA action date of August 21, 2025, for donidalorsen, based on positive Phase 3 and Phase 2 OLE study results.
• Donidalorsen targets prekallikrein (PKK) to reduce HAE attacks and has demonstrated a 96% reduction in attack rates in studies.
FDA Accepts Ionis' Donidalorsen NDA for Hereditary Angioedema Prophylaxis
• The FDA has accepted Ionis Pharmaceuticals' NDA for donidalorsen, an RNA-targeted medicine, for prophylactic treatment of hereditary angioedema (HAE).
• The application is supported by positive Phase 3 OASIS-HAE and OASISplus study results, along with ongoing Phase 2 OLE study data.
• Donidalorsen aims to reduce prekallikrein production, potentially preventing HAE attacks in adult and pediatric patients 12 years and older.
• The FDA has set a PDUFA action date of August 21, 2025, for donidalorsen, which has also received Orphan Drug Designation.
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