EMA Committee Recommends Approval of Ionis’ & AstraZeneca's Wainzua for Amyloidosis

EMA Committee Recommends Approval of Ionis’ & AstraZeneca's Wainzua for Amyloidosis

By, Admin 23 October 2024

EMA committee recommends approval of Ionis’ & AstraZeneca's Wainzua to treat adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis

Overview

Ionis Pharmaceuticals, Inc. announced that Ionis' and AstraZeneca's Wainzua (eplontersen) has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. If approved by the European Commission, Wainzua will be the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector.

NEURO-TTRansform Phase 3 Trial

  • The CHMP based its opinion on the positive NEURO-TTRansform phase 3 trial which showed that through 66 weeks, patients treated with Wainzua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) versus external placebo. 
  • Wainzua continued to demonstrate a favourable safety and tolerability profile throughout the NEURO-TTRansform trial.

The Disease: ATTRv-PN 

  • ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. 
  • Wainzua is a once-monthly RNA-targeted medicine designed to reduce the production of TTR protein at its source.

Words from the CEO: Ionis

  • Hereditary transthyretin-mediated amyloidosis with polyneuropathy remains a progressive and debilitating disease in Europe and other parts of the world, despite currently available medicines,"" said Brett P. Monia, Ph.D., chief executive officer, Ionis. 
  • The CHMP recommendation is an important step toward making Wainzua available in Europe, which, if approved, will be the only medicine in the EU for the treatment of transthyretin-mediated amyloidosis with polyneuropathy that can be self-administered monthly via an auto-injector. We are proud to partner with AstraZeneca whose global leadership and expertise positions our alliance to rapidly and effectively bring Wainzua to many people living with hereditary transthyretin-mediated amyloidosis with polyneuropathy in Europe, pending the EMA's decision.""

Wainua (eplontersen) Approval

  • In December 2023, eplontersen was approved in the US for the treatment of ATTRv-PN, under the brand name Wainua (eplontersen). 
  • Wainua is now gaining approvals in additional countries worldwide, including Canada. 
  • As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing Wainua in the US The companies are seeking regulatory approval in Europe and other parts of the world where AstraZeneca has exclusive rest of world commercialization and development rights. 
  • Eplontersen has also been granted Orphan Drug Designation in the US and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Eplontersen in Treating ATTR-CM

  • Eplontersen is also currently being evaluated in the global CARDIO-TTRansform phase 3 study for the treatment of adults with ATTR-CM. 
  • The trial is fully enrolled with more than 1,400 patients – making it the largest, most comprehensive study to date in this patient population.

ATTR: Pathophysiology

  • ATTR is caused by the accumulation of liver-derived misfolded TTR protein in tissues, such as the heart and the peripheral nerves, causing organ damage and failure. 
  • ATTR then causes complications, leading to cardiovascular, neurological and renal diseases such as heart failure and chronic kidney disease.
  • There are both hereditary (ATTRv) and non-hereditary (wild-type) forms of ATTR. ATTR is a rapidly progressive and fatal disease that requires timely recognition of symptoms. 
  • ATTR has several phenotypes including ATTR-cardiomyopathy (CM), which predominantly impacts the heart, potentially leading to heart failure, ATTR-polyneuropathy (PN), which predominantly affects the peripheral nervous system and mixed phenotype, where patients experience symptoms of both. 
  • Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM and about 10,000 – 40,000 patients with ATTRv-PN.

NEURO-TTRansform Trial

  • NEURO-TTRansform is a global, open-label, randomized trial evaluating the efficacy and safety of eplontersen in patients with ATTRv-PN at week 35, week 66 and week 85. 
  • The final analysis comparing eplontersen to an external placebo group was completed at week 66. 
  • All patients were then followed on treatment until week 85 and evaluated four weeks after the last dose in an end-of-trial assessment. 
  • Following treatment and the end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving eplontersen once every four weeks or enter a 20-week post-treatment evaluation period, which is still ongoing. 
  • Full results from the NEURO-TTRansform trial were published in The Journal of the American Medical Association (JAMA) demonstrating the benefit of eplontersen across the spectrum of ATTRv-PN at 35, 66 and 85 weeks of treatment.

About the Drug: Eplontersen

Eplontersen is a once-monthly RNA-targeted medicine that provides upstream suppression of transthyretin (TTR) production and is designed to precisely target and reduce the production of TTR protein at its source in the liver.

Wainzua Approval in US

Wainzua has been approved in the US and Canada under the brand name Wainua (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN.

Ionis Work on Serious Diseases

  • For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. 
  • Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need. 
  • As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing.

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