hATTR-PN Drug WAINUA™ Available at Orsini Specialty Pharmacy

Ionis Pharmaceuticals, Inc. (Ionis) recently announced that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca’s WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for the treatment of ATTRv-PN that can be self-administered via an auto-injector.

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. WAINUA is a ligand-conjugated antisense oligonucleotide (LICA) medicine designed to reduce the production of TTR protein at its source.

WAINUA enters a competitive space, as market leader Alnylam Pharmaceuticals offers Onpattro (patisiran) and Amvuttra (vutrisiran) as treatments for hATTR-PN. Ionis already markets Tegsedi (inotersen) for the disease. WAINUA will also compete against Pfizer’s Vyndaqel in the transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) market.

WAINUA will be available in the U.S. in January 2024. WAINUA is available at the following the Authorized Specialty Pharmacy Provider (SPP) Orsini Pharmaceutical Services who can provide support to help patients prescribed WAINUA.

As part of a global development and commercialization agreement, AstraZeneca and Ionis will commercialize WAINUA for the treatment of ATTRv-PN in the U.S. and are seeking regulatory approval in Europe and other parts of the world.

This agreement was recently expanded to include exclusive rights for AstraZeneca to commercialize WAINUA in Latin America in addition to all other countries outside the U.S. WAINUA was granted Orphan Drug Designation in the U.S. and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

Providing patients with comprehensive and compassionate care since 1987, Orsini Specialty Pharmacy is a leader in rare diseases and gene therapies. Orsini partners with biopharma innovators, healthcare providers, and payors to support patients and their families in accessing revolutionary treatments for rare diseases.​

Through integrated pharmacy distribution, patient services, clinical management, and convenient home infusion services, Orsini delivers customized solutions that simplify how patients connect to advanced therapies.