Biogen Reports P-II (CELIA) Trial Results on Diranersen in Early Alzheimer’s Disease

Shots: Biogen has reported the global P-II (CELIA) trial data assessing diranersen [60mg (Q24W), 115mg (Q24W), or 115mg (Q12W); intrathecal) vs PBO over 76wks. in 416 pts with mild cognitive impairment due to Alzheimer’s disease (AD) or mild AD dementia Although CELIA did not meet its 1EP of dose response on CDR-SB change from baseline…

ByRidhi RastogiMay 15, 2026

NEWS

Ionis’ Zilganersen Receives US FDA Priority Review for Alexander Disease

Shots: The US FDA has accepted NDA & granted priority review to zilganersen for the treatment of Alexander disease (AxD), with PDUFA target action date of Sep 22, 2026 NDA was backed by the P-III trial assessing zilganersen (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.) for 60wks., followed by an open label…

ByRidhi RastogiMarch 25, 2026

NEWS

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

Shots: The US FDA has accepted sNDA & granted priority review to olezarsen for the treatment of severe hypertriglyceridemia (sHTG), with a PDUFA action date of Jun 30, 2026 sNDA was backed by the P-III (CORE: n=617 & CORE2: n=446) trials assessing olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in sHTG adults…

ByRidhi RastogiFebruary 27, 2026

NEWS

The EC Approves Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Shots: The EC has approved Dawnzera (SC autoinjector; Q4W/Q8W) for the routine prevention of HAE attacks in pts (≥12yrs.) Approval was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate, with 94% overall mean monthly reduction at 1yr. in the OASISplus OLE study Approval…

ByRidhi RastogiJanuary 22, 2026

NEWS

Ionis’ Zilganersen Receives the US FDA’s Breakthrough Therapy Designation for Alexander Disease

Shots: The US FDA has granted BTD to zilganersen for the treatment of Alexander disease (AxD) Designation was supported by the topline P-III trial data, which assessed zilganersen (25 or 50mg) vs control in 54 AxD pts (1.5-53yrs.). Zilganersen (50mg) met the 1EP of gait speed as assessed by 10-Meter Walk Test at 61wks., plus…

ByRidhi RastogiDecember 3, 2025

NEWS

The CHMP Recommends Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Shots: The CHMP has recommended Dawnzera for the routine prevention of HAE attacks in pts (≥12yrs.) following the US FDA approval in Aug 2025; EC’s decision is expected in Q1’26 Opinion was based on P-III (OASIS-HAE) trial & OASISplus study, showing improvements across multiple domains, incl. sustained reduction in mean monthly HAE attack rate even when Dawnzera…

ByRidhi RastogiNovember 17, 2025

NEWS

Ionis Highlights P-III (CORE & CORE2) Trials Results of Olezarsen for Severe Hypertriglyceridemia (sHTG) at AHA 2025

Shots: The P-III (CORE: n=617 & CORE2: n=446) trials assessed olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in sHTG adults; the US FDA’s sNDA filing is expected by 2025 end Both trials met their 1EP with ~72% PBO-adjusted fasting triglyceride (TG) reduction at 6mos., sustained to 12mos., while 89% (50mg) & 88%…

ByRidhi RastogiNovember 10, 2025

NEWS

Ionis’ Tryngolza (Olezarsen) Receives the EC’s Approval for Familial Chylomicronemia Syndrome

Shots: The EC has approved Tryngolza (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome Approval was based on P-III (Balance) trial assessing Tryngolza (Q4W) vs PBO, which showed reduced triglyceride levels at 6mos., sustained through 12mos., with decrease in acute pancreatitis events over 12mos.; data…

ByRidhi RastogiSeptember 23, 2025

NEWS

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…

ByRidhi RastogiSeptember 12, 2025

NEWS

Ionis Reveals Topline P-III (CORE & CORE2) Trial Data of Olezarsen for Severe Hypertriglyceridemia

Shots: Ionis has reported P-III (CORE: n=617 & CORE2: n=446) trials data assessing olezarsen (50 or 80mg, SC, Q4W) vs PBO for 12mos. in adults with severe hypertriglyceridemia (sHTG); the US FDA’s sNDA submission expected by year end Both studies met their 1EP, with CORE showing reduction in fasting triglycerides by 72% (80mg) & 63%…

ByRidhi RastogiSeptember 4, 2025

Biogen Reports P-II (CELIA) Trial Results on Diranersen in Early Alzheimer’s Disease

Ionis’ Zilganersen Receives US FDA Priority Review for Alexander Disease

Ionis’ Olezarsen sNDA Secures the US FDA Priority Review for Severe Hypertriglyceridemia

The EC Approves Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Ionis’ Zilganersen Receives the US FDA’s Breakthrough Therapy Designation for Alexander Disease

The CHMP Recommends Ionis and Otsuka’s Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Ionis Highlights P-III (CORE & CORE2) Trials Results of Olezarsen for Severe Hypertriglyceridemia (sHTG) at AHA 2025

Ionis’ Tryngolza (Olezarsen) Receives the EC’s Approval for Familial Chylomicronemia Syndrome

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Ionis Reveals Topline P-III (CORE & CORE2) Trial Data of Olezarsen for Severe Hypertriglyceridemia

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