NICE backs AZ/Ionis' Wainzua for ATTR polyneuropathy
Just over a month after it was approved in the UK, AstraZeneca and Ionis' Wainzua has been recommended for NHS use in the treatment of patients with polyneuropathy associated with transthyretin amyloidosis (ATTR-PN).
New guidance from health technology assessment (HTA) agency NICE backs the use of Wainzua (eplontersen) to treat stage 1 or 2 polyneuropathy in patients with ATTR, becoming the first option that can be self-administered by patients using a pen-injector, rather than being administered by a healthcare professional.
NICE' has said that Wainzua can be used as an alternative to Alnylam's Amvuttra (vutrisiran), which was recommended for NHS use last year. Amvuttra is dosed every three months via a pre-filled syringe, but needs to be administered by medical staff.
The least expensive of the two drugs should be used, taking into account factors like administration costs, the dose required, and the price per dose, unless it is unsuitable for some reason, according to the new guidance.
Both Wainzua and Amvuttra work by preventing the production of the TTR protein that accumulates in ATTR, causing tissue damage. When it affects the nerves, leading to ATTR-PN, symptoms include pain, discomfort, progressive weakness and loss of sensation in the legs and arms, and mobility difficulties.
The two new therapies should be used instead of Alnylam's older drug Onpattro (patisiran), first cleared for NHS use in 2019, which NICE calculates is now less cost-effective due to the number of vials needed per patient and administration costs.
AZ's list price for Wainzua is around £32,000 for a 45mg vial, equivalent to an annual cost of a little over £383,000, ahead of a confidential discount offered to the NHS. That puts it close to the cost of Amvuttra, priced at approximately £95,900 per 25mg subcutaneous injection, which also works out at £383,000 per year.
Wainzua was approved by UK medicines regulator MHRA in October on the back of the NEURO-TTRansform trial, which showed that Wainzua reduced levels of TTR by 82%, while a placebo group saw an 11% reduction and also reduced the progression of neurological impairment.
The drug is also being developed to treat cardiomyopathy associated with ATTR, and is in a 1,400-patient phase 3 trial (CARDIO-TTRansform), which is due to read out within the next few months.
AZ licensed Wainzua from Ionis in a $3.6 billion deal in 2021, and sees the drug as a future pillar of its cardiovascular, renal, and metabolism (CVRM) business unit.
Some analysts agree that it has strong sales potential – possibly able to bring in $1 billion or more at peak – assuming it also gets approval for the ATTR cardiomyopathy indication.
Photo by Diana Polekhina on Unsplash
