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Ionis Pharmaceuticals’ WAINUA™ (Eplontersen) Secures FDA Approval for Hereditary Transthyretin-Mediated Amyloidosis Treatment

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has achieved a groundbreaking milestone with the U.S. Food and Drug Administration (FDA) granting approval for WAINUA™ (eplontersen) in collaboration with AstraZeneca.

This approval, based on the Phase 3 NEURO-TTRansform study, marks a significant advancement in treating polyneuropathy associated with hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

Key Highlights:

  • Positive Clinical Results: The FDA approval is founded on a positive 35-week interim analysis from the NEURO-TTRansform study. WAINUA demonstrated consistent and sustained benefits, halting neuropathy disease progression, and enhancing both neuropathy impairment and quality of life.
  • Availability: WAINUA, a self-administered medicine through an auto-injector, will be accessible in the U.S. from January 2024.
  • Global Impact: Additional regulatory reviews for WAINUA are underway globally, aiming to expand its availability beyond the U.S.

Scientific Significance:

  • Unique Treatment Approach: WAINUA, a ligand-conjugated antisense oligonucleotide (LICA), acts by inhibiting the production of transthyretin protein at its source.
  • Patient Benefits: The positive outcomes of WAINUA on serum transthyretin concentration, neuropathy impairment, and quality of life provide a promising solution for those living with the relentless effects of ATTRv-PN.

Expert Perspectives:

  • Dr. Michael J. Polydefkis: “Approval of WAINUA represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease.”
  • Brett P. Monia, Ph.D., CEO at Ionis: “WAINUA will be the first in a steady cadence of potential commercial launches for the company.”
  • Isabelle Lousada, President and CEO, Amyloidosis Research Consortium: “Exciting to see new innovations coming through and increased efforts to raise awareness in an area that has often been overlooked or neglected.”

Ruud Dobber, EVP, BioPharmaceuticals Business Unit, AstraZeneca: “U.S. approval of WAINUA offers a new treatment option that provides consistent and sustained reduction in serum TTR concentration while halting disease progression and improving quality of life.”

Global Collaboration and Future Research:

  • Development Agreement: AstraZeneca and Ionis are set to commercialize WAINUA globally, seeking regulatory approvals in Europe and other parts of the world. This collaboration recently expanded to include exclusive rights for AstraZeneca to commercialize WAINUA in Latin America.
  • Ongoing Research: Eplontersen is being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), showcasing its potential to address this fatal condition.

Safety Information:

  • Vitamin A Levels: Reduced serum vitamin A levels may occur; supplementation is recommended.
  • Adverse Reactions: Common adverse reactions include decreased vitamin A levels and vomiting.

The FDA’s approval of WAINUA represents a crucial step forward in addressing the debilitating effects of hereditary transthyretin-mediated amyloid polyneuropathy, offering patients a promising treatment option with the potential for broader global impact.


Joan David-Leonhard

Joan David Leonhard is a recent Pharm.D graduate with a strong passion for the pharmaceutical industry and a particular interest in pharmaceutical media and communication. Her brief internship experience includes roles in pharmacy where she built strong patient-pharmacist relationships and a pharmaceutical media internship where she actively contributed to drug information articles, blog posts, social media engagement, and various media projects.

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