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Orsini selected as the exclusive specialty pharmacy for WAINUA (eplontersen) for adults with hereditary transthyretin-mediated amyloid polyneuropathy

ELK GROVE VILLAGE, IL – Jan. 4, 2024 – Orsini Specialty Pharmacy (“Orsini”) has been selected by AstraZeneca and Ionis as the exclusive specialty pharmacy partner for WAINUATM (eplontersen), an FDA-approved treatment for adults living with hereditary transthyretin-mediated amyloid polyneuropathy (commonly referred to as hATTR-PN or ATTRv-PN). WAINUA  is a subcutaneous injection that can be self-administered, designed to reduce the production of transthyretin and slow the progression of polyneuropathy for people living with ATTRv-PN. Read the US Full Prescribing Information here.

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. For the estimated 40,000 patients worldwide living with ATTRv-PN, WAINUA meets an urgent unmet medical need for new treatment options. Eplontersen is also being evaluated in the CARDIO-TTRansform Phase III trial for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) and has the potential to be a best-in-class treatment across all forms of transthyretin-mediated amyloidosis.

Orsini is proud to be the exclusive specialty pharmacy offering access to WAINUA as a new treatment option for patients living with ATTRv-PN,” Orsini CEO Brandon Tom said. “We are on a mission to compassionately serve rare disease patients, and WAINUA is an important addition to our Neurology Center of Excellence.”

Important Safety Information for WAINUA™ (eplontersen)

  • Warnings and Precautions: Reduced Serum Vitamin A Levels and Recommended Supplementation—WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.
  • Adverse Reactions: Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).
  • Indication: WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Please see link to US Full Prescribing Information for WAINUA.

Note: While Orsini is the exclusive specialty pharmacy partner for WAINUA, it may also be available for use by integrated delivery networks (IDNs) within qualifying IDN-owned specialty pharmacies.

About Orsini Specialty Pharmacy

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. Orsini’s high-touch care model centers on experienced and trained therapy care teams that provide personalized patient care to ensure that No Patient is Left Behind™.

Orsini Specialty Pharmacy holds accreditations with the Accreditation Commission for Health Care (ACHC), The Joint Commission, URAC and NABP. Orsini has earned URAC’s Rare Disease Pharmacy Center of Excellence Designation and ACHC’s Distinction in Rare Diseases and Orphan Drugs. For more information, contact Orsini at 847-734-7373 ext. 505, email us at, or visit