AstraZeneca AZN and Ionis Pharmaceuticals IONS announced that the Committee for Medicinal Products for Human Use (CHMP) has recommended approval for their drug Wainzua (eplontersen) for treating hereditary transthyretin-mediated amyloidosis, commonly called hATTR-PN or ATTRv-PN.

If the European Commission approves Wainzua, it will become the only medicine approved for treating ATTRv-PN that can be self-administered monthly via an auto-injector in the European Union.

Wainzua was approved by the name of Wainua in the United States in December last year. The drug was commercially launched in the United States in the first quarter of 2024.

ATTRv-PN is a debilitating disease, which leads to peripheral nerve damage and affects an estimated 40,000 people worldwide. It can cause motor disability within five years of diagnosis, which can become fatal if not treated properly within a decade.

Year to date, shares of AstraZeneca have increased 15% compared with the industry’s rally of 20.5%.

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Year to date, shares of Ionis have plunged 23% compared with the industry’s decline of 1%.

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CHMP Nod Based on AZN's NEURO-TTRansform Study

The latest CHMP opinion was based on positive data from the phase III NEURO-TTRansform study, which evaluated the safety and efficacy of Wainzua in patients with ATTRv-PN.

Data from the same showed that treatment with Wainzua led to consistent and sustained benefit in serum transthyretin (TTR) concentration and neuropathy impairment as measured by modified Neuropathy Impairment Score +7. This was the study's co-primary endpoint. Treatment with Wainzua also improved quality of life versus placebo, a key secondary endpoint of the NEURO-TTRansform study.

Wainzua is an RNA-targeted medicine designed to reduce the production of TTR protein at its source in the liver to potentially treat all types of transthyretin-mediated amyloidosis.

More Updates on AZN's Wainzua

While Ionis and AstraZeneca jointly market Wainua for ATTRv-PN in the United States, AZN has exclusive rights to commercialize Wainua outside U.S. markets. In the United States, Ionis receives royalties from AstraZeneca on Wainua’s sales.

Besides Europe, AZN and IONS have also submitted regulatory filings seeking approval for eplontersen in ATTRv-PN in several other countries.

AstraZeneca and Ionis are also developing eplontersen for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in the phase III CARDIO-TTRansform study. Data from the same is expected in the first half of 2025.

AZN's Zacks Rank & Other Stocks to Consider

AstraZeneca currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks in the biotech sector are ANI Pharmaceuticals, Inc. ANIP and Elevation Oncology, Inc. ELEV, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.69 to $4.81. Earnings per share estimates for 2025 have improved from $5.37 to $5.86. Year to date, shares of ANIP have risen 7.5%.

ANIP’s earnings beat estimates in each of the trailing four quarters, with the average surprise being 31.32%.

In the past 60 days, estimates for Elevation Oncology’s 2024 loss per share have narrowed from 86 cents to 82 cents. Loss per share estimates for 2025 have narrowed from 90 cents to 86 cents. Year to date, shares of ELEV have increased 9%.

ELEV’s earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 12.05%.

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