Pharmacy Bulletin

Pharmacy Bulletin

We share important prescription drug information to help you stay informed about updates concerning particular prescription medicines.

VativoRx Bottle update

AstraZeneca and Ionis Pharmaceuticals won U.S. Food and Drug Administration (FDA) approval on Dec. 21, 2023, for Wainua (eplontersen) injection. The ligand-conjugated antisense oligonucleotide (LICA) is indicated to treat adults who have peripheral nerve disease (polyneuropathy) due to hereditary transthyretin-mediated amyloidosis (hATTR-PN). Once-monthly 45mg subcutaneous (SC) injections can be given by a healthcare professional, a caregiver, or the patient. Market launch is planned for January 2024, but distribution and pricing information have not yet been announced. Complete prescribing information for Wainua is here.

At a Glance

  • Brand (Generic) Name: Wainua (eplontersen)
  • Manufacturer: AstraZeneca and Ionis
  • Date Approved: Dec. 21, 2023
  • Indication: to treat adults who have hATTR-PN
  • Dosage Forms Available: single-dose pen devices containing 45mg of Wainua
  • Launch Date: January 2024
  • Estimated Annual Cost: Not yet available
  • A group of rare diseases that produces disability, pain, and organ damage, amyloidosis is believed to affect fewer than 200,000 individuals, almost all of them adults, in the United States, according to the Amyloidosis Foundation.
  • hATTR is caused by mutations in the genes that produce a protein essential for the distribution of thyroid hormones and vitamin A.
  • Ordinarily, pieces of messenger ribonucleic acid (mRNA) transfer specific amino acid sequences from genes into cells that then produce the specified proteins. When some of the sequences mutate, abnormal disease-causing proteins sometimes are made.
  • The proteins that cause hATTR amyloidosis are folded and bent in ways the body cannot use, so they build up – mostly in the heart and nerve fibers in the body’s periphery. Eventually, these amyloid fibrils interfere with organ function, leading to death.
  • Patients who have hATTR-PN have nerve damage, which causes pain, tingling, numbness, and weakness, beginning in their feet and gradually spreading to other parts of the body.
  • A variation of the condition that predominantly damages the heart is called cardiomyopathy of hATTR (hATTR-CM). It causes symptoms such as arrhythmias, chest pain, edema, and fatigue.
  • Many patients have symptoms of both hATTR-PN and hATTR-CM, as well as other forms of hATTR and unrelated diseases, which interferes with accurate diagnoses. 
  • By trapping the mRNA of TTR, Wainua decreases circulating fibrils that can infiltrate nerves and other body tissues.
  • In the NEURO-TTR clinical trial, patients treated with Wainua averaged an 81.7% decrease in TTR blood levels as compared to an 11.2% reduction for those using a placebo.
  • Side effects were minimal in the study groups, with vomiting and low blood levels of vitamin A being reported most often.
  • Ionis also produces Tegsedi™ (inotersen) for the treatment of adults who have hATTR-PN. Packaged in single-dose prefilled syringes for SC administration, it decreases the amounts of TTR proteins in the blood by blocking the mutated RNA that produces them. A boxed warning cautions that using it may cause thrombocytopenia (possibly sudden and dangerous drops in platelet count) and/or glomerulonephritis (kidney inflammation that may cause kidney failure). Due to its potentially severe side effects, dispensing of Tegsedi is limited through a risk evaluation and mitigation strategy (REMS).
  • Onpattro® (patisiran – Alnylam) lipid complex injection is a small interfering ribonucleic acid (siRNA) indicated for treating adults who have hATTR-PN. Onpattro blocks (silences) mutated RNA that causes hATTR. It is given by intravenous (IV) infusion once every three weeks.
  • Pfizer’s Vyndaqel® (tafamidis meglumine) capsules and Vyndamax (tafamidis) capsules treat ATTR-CM by stabilizing TTR, which decreases the formation and deposit of amyloid fibrils. Either form of the drug is taken once daily.
  • Wainua has Orphan Drug status for treating transthyretin-mediated amyloidosis.