Ionis Pharmaceuticals and AstraZeneca are wrapping up the year with a much-anticipated present from the FDA: an approval for the companies’ transthyretin amyloidosis (ATTR) drug Wainua.
More specifically, the agency signed off on the ligand-conjugated antisense oligonucleotide (LICA) drug to treat polyneuropathy in adult patients with hereditary transthyretin-mediated amyloidosis (ATTRv-PN), a rare and often fatal disease that affects 40,000 people globally, the companies said in a press release.
The nod was backed by phase 3 data showing Wainua's ability to provide “consistent and sustained” benefit in outcomes measures, including serum transthyretin concentration and neuropathy impairment, as well as improvements in quality of life.
Crucially, Wainua comes in an auto-injector form that patients can administer themselves once a month. That gives the treatment a convenience edge against the competition, including Alnylam’s Onpattro, which is delivered subcutaneously at treatment centers.
Despite the Alnylam option, the unmet need for the genetic disease remains “very, very high” Ionis’ CEO Brett Monia, Ph.D., said in an interview with Fierce Pharma. More than 80% of patients with the disease are currently untreated, Monia said, due to a general lack of disease awareness and a high prevalence of misdiagnoses.
AstraZeneca joined in on the drug in 2021 with a $200 million upfront payment that got the British pharma exclusive ex-U.S. commercialization rights. Under the deal, AZ manufactures the commercial supply and will pay Ionis up to $485 million in development and approval milestones, plus up to $2.9 billion in sales-relates milestones.
Marrying Ionis’ long-held expertise in amyloidosis and rare diseases plus AZ’s experience in cardiovascular diseases and global foothold enables the two to reach “as many patients as possible,” Monia said. Meanwhile, for AstraZeneca, the approval marks the start of a “very exciting journey” in the amyloidosis field, executive vice president and president of AZ’s biopharmaceuticals business unit, Ruud Dobber, Ph.D., noted in an interview.
The Wainua collaboration isn’t stopping here. Next up for the drug is a planned label expansion bid for transthyretin amyloid cardiomyopathy (ATTR-CM), which affects some 400,000 to 500,000 patients globally. The companies are already testing out the med in what Monia calls the “largest study ever conducted” in ATTR-CM. That trial is expected to read out in 2025, Dobber said.
In the smaller ATTRv-PN population, Wainua's competition includes Alnylam’ Onpattro, which was approved in 2018 as the first med for the condition. While Alnylam had the same ATTR-CM hopes as Ionis and AZ, the company gave up on the label expansion after the FDA denied its approval earlier this year.
Meanwhile, Pfizer’s blockbuster tafamidis (which is sold as Vyndamax and Vyndaqel) was the first in the ATTR-CM space with its 2019 approval. Pfizer’s first-to-market options might be “difficult to displace” and seem “quite entrenched,” Evercore ISI analysts noted in September when Alnylam looked poised for an ATTR-CM expansion
Ionis’ drug could rake in $1.3 billion in 2030 sales, SVB Leerink analyst Mani Foroohar wrote in a 2021 note to clients. The companies are prepared to launch Wainua in the U.S. in January, Monia said.
Wainua represents the first in a "steady cadence" of potential commercial launches for Ionis, the CEO said in the press release. The company's familial chylomicronemia syndrome (FCS) candidate olezarsen could launch as soon as the second half of next year, according to Monia, which would mark Ionis' first solo launch.

https://www.pharma.com/pharma/ionis-astrazeneca-kick-wainua-journey-rare-disease-transthyretin-amyloidosis-fda-approval