Ionis and AstraZeneca drug gains FDA approval for Hereditary Transthyretin Amyloidosis

Ionis Pharmaceuticals and AstraZeneca are capping off the year with a notable achievement—a coveted FDA approval for their transthyretin amyloidosis (ATTR) drug, Wainua. This regulatory green light specifically encompasses the ligand-conjugated antisense oligonucleotide (LICA) medication, designed to combat polyneuropathy in adult patients grappling with hereditary transthyretin-mediated amyloidosis (ATTRv-PN).

 This rare and often fatal disease impacts a global population of 40,000 individuals, as highlighted in a joint press release disseminated by the pharmaceutical collaborators.

The FDA’s nod was underpinned by robust phase 3 data showcasing Wainua’s consistent and sustained efficacy across various outcome metrics. These encompassed significant improvements in serum transthyretin concentration, reductions in neuropathy impairment, and noteworthy enhancements in the overall quality of life for the patients.

What sets Wainua apart is its user-friendly auto-injector format, empowering patients to administer the treatment every month independently. This feature confers a distinct advantage over Alnylam’s Onpattro, necessitating subcutaneous delivery at specialized treatment centers.

Despite the existence of alternatives such as Onpattro, the unmet needs for this genetic disease persist at alarming levels. According to Brett Monia, Ph.D., Ionis’ CEO, over 80% of patients with the disease remain untreated, owing to a combination of insufficient disease awareness and a prevalence of misdiagnoses.

AstraZeneca entered into this collaboration in 2021, offering a substantial upfront payment of $200 million to secure exclusive ex-U.S. commercialization rights. Under the agreement, AstraZeneca is responsible for the commercial supply, committing to payments of up to $485 million in development and approval milestones, along with potential sales-related milestones totaling up to $2.9 billion.

The collaborative synergy between Ionis’ extensive expertise in amyloidosis and rare diseases and AstraZeneca’s prowess in cardiovascular diseases, coupled with its global reach, positions the two entities to cater to a broader patient demographic. Monia expressed confidence that this collaboration would enable them to reach “as many patients as possible.” Meanwhile, AstraZeneca views the approval as the inception of an “exciting journey” in the amyloidosis field, according to Ruud Dobber, Ph.D., executive vice president and president of AstraZeneca’s biopharmaceuticals business unit.

The journey for Wainua doesn’t culminate with this approval. The next phase involves a meticulously planned label expansion to address transthyretin amyloid cardiomyopathy (ATTR-CM), affecting an estimated 400,000 to 500,000 patients globally. Ionis and AstraZeneca are currently conducting what Monia describes as the “largest study ever conducted” in ATTR-CM, with results expected in 2025.

In the realm of ATTRv-PN, Wainua contends primarily with Alnylam’s Onpattro, which received approval in 2018 as the pioneer medication for this condition. While both Ionis and Alnylam initially harbored aspirations for an ATTR-CM expansion, Alnylam abandoned these hopes after the FDA denied approval earlier this year.

Pfizer’s tafamidis, marketed as Vyndamax and Vyndaqel, secured approval in 2019, making it the trailblazer in the ATTR-CM space. Analysts have underscored that Pfizer’s early market entry may pose challenges for competitors, characterizing the options as “difficult to displace” and appearing “quite entrenched.”

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