Alexion Pharmaceuticals, Inc.

A year after the FDA rejected full approval of Andexxa (andexanet alfa), AstraZeneca has decided to voluntarily withdraw the anticoagulant reversal drug from the US citing commercial reasons. The regulator indicated that the company will end commercial sales of Andexxa by December 22, and the product will no longer be manufactured or sold in the US after this date.Granted accelerated approval back in 2018 as an antidote for the reversal of Factor Xa inhibitors, AstraZeneca gained Andexxa in 2021 through its $39-billion purchase of Alexion, which itself acquired the drug from Portola Pharmaceuticals a year earlier for $1.4 billion. Last January, AstraZeneca submitted a supplemental biologics application seeking to convert the accelerated approval into a full approval. However, the FDA rejected the filing and issued a complete response letter — at the time, AstraZeneca told FirstWord that "while discussions with the FDA are ongoing, Andexxa will remain on the US market."The FDA has now said that based on available data, the serious risks associated with Andexxa, including the increase in thromboembolic events, outweigh its benefits. The agency noted that in the ANNEXA-I trial — which was designed to verify the clinical benefit of Andexxa among patients with intracerebral haemorrhage — AstraZeneca's therapy was associated with a doubling in the rate of thromboses and thrombosis-related deaths at day 30 versus usual care.According to the FDA, the incidence of thrombosis was 14.6% in the Andexxa arm, compared to 6.9% for usual care, while thrombosis-related deaths occurred at rates of 2.5% and 0.9%, respectively.

The FDA said it believes the risks of Andexxa outweigh its benefits. AstraZeneca is responding by pulling the medicine from the market. Seven years after the FDA's accelerated approval of bleeding reversal agent Andexxa, which was then under the ownership of Portola Pharmaceuticals, the drug's round trip on the U.S. market is coming to an end. Andexxa, now under AstraZeneca's stewardship, is slated to be pulled from the U.S. market next week, according to a Dec. 18 update from the FDA. In the FDA communication, the agency notes that it has "received postmarketing safety data on thromboembolic events, including serious and fatal outcomes, in patients treated with Andexxa." Based on the available data, the agency "considers the risks of the product to outweigh its benefits," it said.The FDA has shared that position with AstraZeneca, and the company "submitted a request to voluntarily withdraw the BLA for the product for commercial reasons," the agency said.Andexxa won its original approval as a treatment for patients on the popular blood thinners Eliquis and Xarelto who needed anticoagulation due to life-threatening or uncontrolled bleeding, with a boxed warning describing the risk of thromboembolic events.While AstraZeneca in 2023 touted the efficacy results of a postmarketing study, that trial recorded a doubling in the rate of thromboses and thrombosis-related deaths at day 30 for patients who received Andexxa versus those who received standard care, according to the FDA.An FDA advisory panel last year scrutinized the drug's postmarketing data, and the agency later rejected AstraZeneca's application to convert the accelerated approval into a full nod. At that time, AstraZeneca said it planned to keep the drug on the market.On Friday, an AstraZeneca spokesperson said the company is "confident that Andexxa has a positive benefit risk profile for eligible patients with life-threatening or uncontrolled bleeds.""In discussions with the FDA, alignment on a feasible path to convert Andexxa from accelerated to traditional approval in the U.S. could not be reached," the spokesperson added. Portola scored accelerated approval for the drug in 2018 after a pair of prior regulatory setbacks. Rare disease drugmaker Alexion scooped up Portola for $1.4 billion in 2020, and AstraZeneca came in afterward with its massive Alexion buyout. Last year, AstraZeneca reported full-year Andexxa sales of $219 million.

Verge Genomics’ sole clinical-stage drug has failed, and the California biotech will shift to a partnership model for its AI-driven discovery platform, Endpoints News has learned. The biotech’s VRG50635 did not meet the bar for efficacy in a Phase 1b in patients with amyotrophic lateral sclerosis, Verge CEO Alice Zhang said in an emailed statement on Tuesday. She said the biotech will publish the full study results in the first half of next year. The trial was completed “as designed,” Zhang said, but it was terminated, according to an update posted to the US federal trials database on Monday. With that, the decade-old biotech will “focus solely” on its AI platform that uses a “proprietary multi-modal molecular and clinical dataset derived directly from patient tissue,” Zhang said. She said there’s been increased demand for its datasets and that the shift will help partners “improve pipeline success rates.” The decision marks a key pivot for the biotech, which formed in 2015 with ambitions to create new medicines based on human genomics. Verge had broad plans for its pipeline, with at least 15 different diseases listed on its website. It was conducting research in neurodegenerative disorders, metabolic conditions, immunology and rare disease. A CD38 enzyme inhibitor for weight management was slated to enter the clinic earlier this year, but doesn’t appear to have done so. Zhang said the move positions Verge to continue investing in its “AI, product, and engineering capabilities,” and will lead to a “leaner cost structure” and extended cash runway. More details about the strategic shift, including updates on Verge’s pipeline and “key hires,” will be disclosed in early 2026, Zhang said. Most of Verge’s C-suite has left in the past 20 months, with chief financial, scientific and medical officers all departing, according to their LinkedIn profiles. President and CBO Jane Rhodes left last year to become CEO of neuro biotech AstronauTx. Verge has not announced a financing round since a $98 million Series B in December 2021. Since then, though, it has forged partnerships with Ferrer and AstraZeneca’s Alexion . Shortly before the Series B, it teamed up with Eli Lilly . Ferrer and Verge outlined a collaboration on VRG50635 last year. The Spanish pharma and Verge said in March 2024 they would co-develop the small molecule PIKfyve inhibitor in Europe, Central and South America, Southeast Asia and Japan. The status of that collaboration, following the trial termination, is unclear. A Ferrer spokesperson didn’t respond to an inquiry from Endpoints.