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Ionis
NEWS

Ionis Reports the US FDA’s Approval of Dawnzera (Donidalorsen) as a Prophylactic Treatment of Hereditary Angioedema (HAE)

Shots: The US FDA has approved Dawnzera (donidalorsen; 80mg SC, Q4W/Q8W) as a prophylactic treatment against attacks in pts (≥12yrs.) with hereditary angioedema; available in the US in coming days Approval was based on P-III (OASIS-HAE) trial & OASISplus OLE study, where P-III showed 81% fewer monthly HAE attacks over 24wks. (87% from 2nd dose) &…
August 22, 2025

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Ionis
NEWS

Ionis Reports the CHMP’s Positive Opinion on Tryngolza for Familial Chylomicronemia Syndrome

Shots: The CHMP has recommended Tryngolza (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome; EC’s decision is expected in Q4’25 Opinion was based on P-III (Balance) trial assessing (Q4W) vs PBO, which showed reduced triglyceride levels at 6mos., sustained through 12mos., with decrease in acute…
July 29, 2025

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Ionis & Sobi
NEWS

Ionis Partners with Sobi for Olezarsen to Treat Familial Chylomicronemia Syndrome (FCS) & Severely Elevated Triglycerides

Shots: Ionis has granted Sobi global exclusive rights of olezarsen excl. the US, Canada & China to treat familial chylomicronemia syndrome (FCS) & severely elevated triglycerides; Ionis to independently market Olezarsen in the US, whereas Theratechnologies holds the rights in Canada As per the deal, Ionis will get an upfront, with potential milestone payments &…
March 27, 2025

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AstraZeneca & Ionis
NEWS

AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the EC’s Approval to Treat Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

Shots: The EC has approved Wainzua to treat ATTRv-PN (stage 1/2 polyneuropathy) in adults pts based on P-III (NEURO-TTRansform) trial assessing eplontersen vs external PBO over 66wks., with follow-up to Wk. 85 & an end-of-trial evaluation. Eligible pts could then enter an ongoing OLE study Trial showed sustained benefits in co-1EPs of serum transthyretin…
March 11, 2025

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Ionis
NEWS

Ionis’ Tryngolza (Olezarsen) Receives the US FDA’s Approval to Treat Adults with Familial Chylomicronemia Syndrome (FCS) 

Shots:    The US FDA has approved Tryngolza (80mg, QM, self-administered via an auto-injector) as an adj. for reducing triglycerides (TG) in FCS patients, based on a P-III (BALANCE) study. Further submission is under review in the EU, with more filings planned   The P-III study involved FCS patients with fasting TG levels of ≥880mg/dL. Trial showed…
December 19, 2024

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EMA Marketing Authorization of New Drugs in October 2024
INSIGHTS+

EMA Marketing Authorization of New Drugs in October 2024

Shots:    The EMA’s CHMP has granted positive opinions to 5 Biologics and 1 New Chemical Entity in October 2024, leading to treatments for patients and advances in the healthcare industry   The major highlighted drugs were Novo Nordisk’s Alhemo to treat Haemophilia A or B with inhibitors and AstraZeneca & Ionis’ Wainzua for Hereditary Transthyretin-Mediated Amyloidosis  PharmaShots has…
November 21, 2024

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AstraZeneca & Ionis
NEWS

AstraZeneca and Ionis’ Wainzua (Eplontersen) Receives the CHMP’s Positive Opinion to Treat Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy 

  Shots:    The opinion of Wainzua for ATTRv-PN (stage 1/2 polyneuropathy) was supported by its P-III (NEURO-TTRansform) study vs external PBO for over wk.66, with a follow-up until wk.85 & an end-of-trial evaluation. Eligible patients could then enter an ongoing OLE study  Study depicted sustained benefits in co-1EPs of serum transthyretin (TTR) levels & neuropathy…
October 21, 2024

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