Veppanu, the first PROTAC therapy approved by the FDA, improved progression free survival by 43% versus AstraZeneca’s Faslodex but showed no such significant benefit in the intention-to-treat analysis.

The FDA on Friday greenlit Pfizer and Arvinas’ protein degrader Veppanu for the treatment of certain types of breast cancer as the first-ever PROTAC therapy. Results from the Phase 3 VERITAC-2 study paved the way for Veppanu’s approval, despite mixed topline data in March last year.

Short for proteolysis-targeting chimeras, these drugs actively target and destroy disease-causing molecules. Veppanu, formerly known as vepdegestrant, is specifically indicated for patients with advanced or metastatic ER-positive and HER2-negative breast cancer who also harbor mutations in the ESR1 gene. Only those who have progressed after a prior line of endocrine therapy are eligible for Veppanu.

As a PROTAC, Veppanu is a small-molecule drug that has two different functional sites, one of which binds to the estrogen receptor and the other to a protein that can trigger the cell’s targeted degradation pathway. This structure leads to widespread degradation of estrogen receptors, in turn disrupting cancer cells’ ability to proliferate.

Pfizer and Arvinas did not present specific VERITAC-2 data in March, only noting that the drug elicited a “statistically significant and clinically meaningful” progression-free survival (PFS) benefit versus AstraZeneca’s Faslodex. However, in the intent-to-treat (ITT) analysis, which covers all patients who were randomized, Veppanu showed no significant PFS effect.

A follow-up readout in June that year showed a 43% reduction in the risk of disease progression or death in patients on Veppanu versus Faslodex. The ITT analysis again failed to hit statistical significance. Overall survival data was immature at the time of filing, according to Arvinas’ Friday release.

As for safety, Veppanu’s label does not carry a boxed warning but does alert patients and prescribers to heart rhythm abnormalities and embryo-fetal toxicities.

Veppanu was originally developed by Arvinas. Pfizer bought into the drug in July 2021 with $650 million upfront, a $350 million equity investment and the promise of up to $1.4 billion in milestones.

It is yet unclear when Veppanu will hit U.S. shelves—Pfizer and Arvinas will first need to select a third-party commercialization partner for the drug. The companies say they are “on track” to identify this partner but have not yet provided a specific timeline.

Veppanu marks Arvinas’ first FDA approval, though there remain “questions around commercialization given ARVN has not yet announced a partner,” analysts at Truist Securities wrote to investors in a Friday note. The firm forecasts $1.1 billion in peak sales for the drug in 2036.

CEO Stéphane Bancel expressed confidence in Moderna’s first quarter, touting the revenue numbers as a sign of the company’s return to prosperity.

After a tussle with the FDA earlier this year over the acceptance of an mRNA flu vaccine for review, Moderna is leaning on international sales and regulatory approvals to support growth for now. The company reported $389 million in revenue for the first quarter of the year, 50% more than what analysts expected.

But Moderna still recorded a major loss of $1.3 billion for the quarter, primarily due to charges to settle litigation with Arbutus involving a patent for certain lipid nanoparticle delivery technology the companies use. This value came in under analyst expectations of $1.61 billion in net losses, even with the litigation charge.

CEO Stéphane Bancel expressed confidence in the quarter, touting the revenue numbers as a sign of the company’s return to prosperity. The $389 million is 260% more than the $108 million recorded for the same period a year ago.

In particular, vaccines sold $352 million, more than the consensus expectation of $223 million, William Blair wrote Friday morning.

Analysts did not get an update on Moderna’s breakeven expectations, which has been a constant theme on recent earnings calls. Before being hit by a number of high-profile setbacks, the company had expected to be in the black by 2028.

In February, the FDA initially refused to review the mRNA-1010 flu vaccine. That situation has been resolved with the agency now accepting the application and granting a decision date of August 5. But Moderna may well need to revise its breakeven guidance.

“We believe recent U.S. regulatory setbacks for mRNA-1010 (flu vaccine) and potentially mRNA-1083 (combo flu/COVID) could pressure this prior guidance, although there does now appear to be a path forward for mRNA-1010 with a PDUFA date of August 5, 2026,” William Blair wrote.

Still, Moderna has $1.9 billion in cash on hand as of March 31, according to the earnings report. Analysts wondered if executives might strike a deal to bolster the company’s offerings. Bancel reiterated his previous statement that Moderna is focused on building the best mRNA platform. But the company has been interested in other complimentary modalities of late, including a T cell engager called mRNA-2808 that advanced into Phase 1/2 testing late last year.

“We’re very focused on expanding to new modalities to enable new families of medicine,” Bancel said. “We are continuing to look at science across the board, whether it’s from academic labs or from companies, public or private.”

One thing Moderna does not need, thanks to its prolific platform, is more programs to work on, Bancel said. Especially as the company has been cutting back on research priorities and trying to reach the breakeven point.

“If we find the right opportunity to increase what we can do, we will, of course, execute on those priorities, but we don’t have a pipeline problem like most companies in the industry. We have an abundance of products,” Bancel said.

Moderna’s net loss for the quarter would have been lower had it not been for the settlement with Arbutus. In February, a federal judge threw out Moderna’s defenses against a patent infringement case that has been brought by Arbutus.

The smaller biotech argues in the case that Moderna accessed its vaccine delivery technology via a sub-license from a third party but never entered into a license agreement to cover the usage. Moderna has argued that Arbutus’ patents are invalid due to obviousness. But the courts and patent office have not agreed and Moderna has to pay $950 million to settle the matter.

This charge did not impact Moderna’s cash for the quarter but litigation-related expenses dogged earnings for the period to the tune of $895 million. The actual payout to Arbutus will occur in the third quarter.

Absent the litigation charges, Moderna’s net loss would have been about $500 million, Bancel said.

Moderna still expects to deliver 10% revenue growth this year while reducing operating expenses. Jefferies noted that the company has leverage to do so through discretionary cuts and AI-driven savings.

The new plant will give Novartis end-to-end capabilities centered on North Carolina, where it plans to have five facilities across three sites.

Novartis has unveiled the seventh and final facility being built under its $23 billion U.S. expansion drive, setting out plans to construct a drug substance plant in North Carolina.

The planned 56,200-square-foot facility in Morrisville, North Carolina, will make active pharmaceutical ingredients (APIs) for solid dosage tablets, capsules and RNA therapeutics. In its Thursday announcement, Novartis called the production site “a critical step” that will strengthen its end‑to‑end U.S. manufacturing capabilities from APIs through finished medicines.

For the first time in its history, Novartis will be equipped to perform end‑to‑end manufacturing for all its advanced technology platforms in the U.S., the company said. The capabilities are part of a push to make all key Novartis medicines for U.S. patients in the U.S.

Novartis unveiled its U.S. expansion strategy one year ago, committing $23 billion to the construction of seven facilities amid President Donald Trump’s threats to impose tariffs on the pharma industry. North Carolina is among the main beneficiaries of Novartis’ investment.

In November, Novartis outlined plans to create a manufacturing hub in the state. The company signaled its intent to build two facilities at a new site in Durham dedicated to manufacturing biologics and sterile packaging, and construct one plant at a new site in Morrisville to make solid dosage tablets and capsules, including packaging.

Novartis is building the newly disclosed API plant on the Morrisville site, which the company expects to open in 2028. The site will support therapies across oncology, immunology, neuroscience, cardiovascular, renal and metabolic disease.

With the API plant adding to its footprint in North Carolina, the drugmaker plans to have five facilities across three sites in the state once it completes its construction program. Novartis is also expanding an existing facility in Durham to support the sterile filling of biologics into syringes and vials. Last year, Novartis said it expected to create 700 jobs in the state by 2030.

Novartis’ expansion in North Carolina is part of an industry-wide wave of investment in the state. In April, AbbVie selected North Carolina as the location for its largest-ever capital investment in one campus. The drugmaker’s $1.4 billion commitment to a plant in Durham followed investments by other companies, including Amgen, Johnson & Johnson, Merck and Roche.

As well as expanding in North Carolina, Novartis has started building radioligand therapy plants in Florida and Texas. The new plants will support a facility that Novartis opened in California last year and sites in Indiana and New Jersey that are undergoing expansions.

The new plant will give Novartis end-to-end capabilities centered on North Carolina, where it plans to have five facilities across three sites.
Novartis has unveiled the seventh and final facility being built under its $23 billion U.S. expansion drive, setting out plans to construct a drug substance plant in North Carolina.

The planned 56,200-square-foot facility in Morrisville, North Carolina, will make active pharmaceutical ingredients (APIs) for solid dosage tablets, capsules and RNA therapeutics. In its Thursday announcement, Novartis called the production site “a critical step” that will strengthen its end‑to‑end U.S. manufacturing capabilities from APIs through finished medicines.

For the first time in its history, Novartis will be equipped to perform end‑to‑end manufacturing for all its advanced technology platforms in the U.S., the company said. The capabilities are part of a push to make all key Novartis medicines for U.S. patients in the U.S.

Novartis unveiled its U.S. expansion strategy one year ago, committing $23 billion to the construction of seven facilities amid President Donald Trump’s threats to impose tariffs on the pharma industry. North Carolina is among the main beneficiaries of Novartis’ investment.

In November, Novartis outlined plans to create a manufacturing hub in the state. The company signaled its intent to build two facilities at a new site in Durham dedicated to manufacturing biologics and sterile packaging, and construct one plant at a new site in Morrisville to make solid dosage tablets and capsules, including packaging.

Novartis is building the newly disclosed API plant on the Morrisville site, which the company expects to open in 2028. The site will support therapies across oncology, immunology, neuroscience, cardiovascular, renal and metabolic disease.

With the API plant adding to its footprint in North Carolina, the drugmaker plans to have five facilities across three sites in the state once it completes its construction program. Novartis is also expanding an existing facility in Durham to support the sterile filling of biologics into syringes and vials. Last year, Novartis said it expected to create 700 jobs in the state by 2030.

Novartis’ expansion in North Carolina is part of an industry-wide wave of investment in the state. In April, AbbVie selected North Carolina as the location for its largest-ever capital investment in one campus. The drugmaker’s $1.4 billion commitment to a plant in Durham followed investments by other companies, including Amgen, Johnson & Johnson, Merck and Roche.

As well as expanding in North Carolina, Novartis has started building radioligand therapy plants in Florida and Texas. The new plants will support a facility that Novartis opened in California last year and sites in Indiana and New Jersey that are undergoing expansions.

CEO Stéphane Bancel expressed confidence in Moderna’s first quarter, touting the revenue numbers as a sign of the company’s return to prosperity.

After a tussle with the FDA earlier this year over the acceptance of an mRNA flu vaccine for review, Moderna is leaning on international sales and regulatory approvals to support growth for now. The company reported $389 million in revenue for the first quarter of the year, 50% more than what analysts expected.

But Moderna still recorded a major loss of $1.3 billion for the quarter, primarily due to charges to settle litigation with Arbutus involving a patent for certain lipid nanoparticle delivery technology the companies use. This value came in under analyst expectations of $1.61 billion in net losses, even with the litigation charge.

CEO Stéphane Bancel expressed confidence in the quarter, touting the revenue numbers as a sign of the company’s return to prosperity. The $389 million is 260% more than the $108 million recorded for the same period a year ago.

In particular, vaccines sold $352 million, more than the consensus expectation of $223 million, William Blair wrote Friday morning.

Analysts did not get an update on Moderna’s breakeven expectations, which has been a constant theme on recent earnings calls. Before being hit by a number of high-profile setbacks, the company had expected to be in the black by 2028.

In February, the FDA initially refused to review the mRNA-1010 flu vaccine. That situation has been resolved with the agency now accepting the application and granting a decision date of August 5. But Moderna may well need to revise its breakeven guidance.

“We believe recent U.S. regulatory setbacks for mRNA-1010 (flu vaccine) and potentially mRNA-1083 (combo flu/COVID) could pressure this prior guidance, although there does now appear to be a path forward for mRNA-1010 with a PDUFA date of August 5, 2026,” William Blair wrote.

Still, Moderna has $1.9 billion in cash on hand as of March 31, according to the earnings report. Analysts wondered if executives might strike a deal to bolster the company’s offerings. Bancel reiterated his previous statement that Moderna is focused on building the best mRNA platform. But the company has been interested in other complimentary modalities of late, including a T cell engager called mRNA-2808 that advanced into Phase 1/2 testing late last year.

“We’re very focused on expanding to new modalities to enable new families of medicine,” Bancel said. “We are continuing to look at science across the board, whether it’s from academic labs or from companies, public or private.”

One thing Moderna does not need, thanks to its prolific platform, is more programs to work on, Bancel said. Especially as the company has been cutting back on research priorities and trying to reach the breakeven point.

“If we find the right opportunity to increase what we can do, we will, of course, execute on those priorities, but we don’t have a pipeline problem like most companies in the industry. We have an abundance of products,” Bancel said.

Moderna’s net loss for the quarter would have been lower had it not been for the settlement with Arbutus. In February, a federal judge threw out Moderna’s defenses against a patent infringement case that has been brought by Arbutus.

The smaller biotech argues in the case that Moderna accessed its vaccine delivery technology via a sub-license from a third party but never entered into a license agreement to cover the usage. Moderna has argued that Arbutus’ patents are invalid due to obviousness. But the courts and patent office have not agreed and Moderna has to pay $950 million to settle the matter.

This charge did not impact Moderna’s cash for the quarter but litigation-related expenses dogged earnings for the period to the tune of $895 million. The actual payout to Arbutus will occur in the third quarter.

Absent the litigation charges, Moderna’s net loss would have been about $500 million, Bancel said.

Moderna still expects to deliver 10% revenue growth this year while reducing operating expenses. Jefferies noted that the company has leverage to do so through discretionary cuts and AI-driven savings.

Foundayo became available on April 9 and has already reached 20,000 patients as Eli Lilly builds its marketing machine for the weight loss pill.

Over 20,000 patients have received Eli Lilly’s oral weight loss pill Foundayo since its April 9 launch. While analysts didn’t get the exact sales figures they were looking for when the pharma giant reported earnings on Thursday, the early launch details seemed to satisfy the hungry hordes.

“Orals are expanding the market vs. cannibalizing injectable share,” BMO Capital Markets wrote Thursday morning.

Indeed, so far 80% of prescriptions have been to patients who have not taken one of this new class of weight loss medications before, Lilly said. And the company hasn’t even begun full scale consumer promotion yet—that is slated for the third quarter, according to the company’s first quarter earnings presentation.

Foundayo has already been added to more than 12 major telehealth platforms, which have accounted for about 35% of volume, Lilly said. About 45% of volume has come via Lilly’s direct-to-consumer platform, LillyDirect, Ilya Yuffa, president of Lilly USA and global customer capabilities, said on Thursday’s call with investors.

Foundayo has collected 8,000 prescribers—a third of whom had never signed a prescription for an oral GLP-1 before, according to Yuffa.

“What we’re hearing is really positive on the overall efficacy and kind of the no hassle factor on a daily oral GLP-1,” he said.

To promote the therapy, Lilly has been working on direct-to-consumer promotion, digital and social media and more ahead of the full-court press planned for later this year. Yuffa said the goal is to improve physician familiarity before the full marketing machine turns on.

While Lilly is able to capitalize on the massive public interest in weight loss, Ricks said Foundayo is different than Zepbound (the weight loss brand name for tirzepatide, which is also marketed as Mounjaro for diabetes). For the Zepbound launch, Lilly was able to tap into familiar prescribers and recognition of the molecule itself.

Rival Novo Nordisk has done the same with its semaglutide franchise, which includes Ozempic for diabetes and the Wegovy injectable or pill for weight loss.

“The three major products that are used in obesity in the United States are all line extensions,” Ricks said. “So the molecule was on the market before, and in some cases, the brand name was used before.”

Ricks noted that in contrast, Foundayo is completely new to the market and physicians. “We’re starting from a much lower baseline. We’ve got to build that, but we’re hugely confident we’ll be able to build it.”

Lilly is also putting its firepower behind international launches at a scale rarely seen. Typically, the U.S. market is accessed first while other smaller markets follow slowly. With Foundayo, Ricks said the company is already working on 40 international regulatory applications.

“We expect that to happen also in one of the most accelerated cadences, perhaps in the history of the industry,” Ricks said.

Lilly will also file for FDA approval of Foundayo for diabetes later this quarter after the drug reduced cardiovascular and death risk in the Phase 3 ACHIEVE-4 trial. The company is testing Foundayo in six additional Phase 3 clinical trials in other diseases.

For earnings, Lilly recorded a massive beat over analyst consensus and raised guidance for the year. The company took home $19.8 billion for the first quarter, which was a 56% increase over the same period in 2025. Key products were, of course, Mounjaro and Zepbound, which contributed mightily to $13.4 billion in revenue for Lilly’s key products.

“After a weaker start to 2026, 1Q results firmly put concerns to bed, with strength across the business,” BMO wrote.

Lilly now expects revenue of $82 billion to $85 billion for the year instead of $80 billion to $83 billion. Earnings per share are expected to fall between $35.50 and $37 instead of $33.50 and $35.

Strong growth in immunology and neurology prompted AbbVie to raise its 2026 outlook and consider future M&A from a position of “ample financial capacity.”

AbbVie recorded $15 billion in revenue for the first quarter, with its immunology portfolio accounting for nearly half of that number.

Global net revenues from the company’s immunology portfolio—buoyed by heavyweights Skyrizi and Rinvoq, rose 16.4% year-over-year, to reach $7.29 billion, according to a Wednesday earnings release. Overall, AbbVie’s first quarter revenues exceeded Q1 2025 by 12.4%.

AbbVie’s shares were trading up just over 3% to $203.89 at the time of publication.

In a note to investors before the opening bell on Wednesday, William Blair noted that the company’s shares “are up slightly in premarket trading with some investor relief” due to the quarterly consensus beat for Skyrizi and Rinvoq and raised overall guidance for 2026. However, AbbVie’s shares are still down over 10% year-to-date, the firm said, “and we believe investors will continue to be focused on market share dynamics for Skyrizi versus competition,” particularly Tremfya in inflammatory bowel disease.

The regulatory submission of a subcutaneous induction regimen for Skyrizi in Crohn’s disease this week “should help alleviate some investor concerns,” William Blair added.

The Illinois pharma’s push to be known beyond its immunology efforts was supported by a 26% revenue jump from its neuroscience portfolio, which achieved $2.87 billion last quarter. In particular, the once-daily, oral migraine med Qulipta brought in $296 million, an increase of 53.6% on a reported basis.

The newest quarterly financials prompted AbbVie to adjust its guidance for the entire year, raising expected adjusted diluted earnings per share for 2026 from a range of $13.96 to $14.16 to $14.08 to $14.28.

While AbbVie has zeroed in on diversifying its portfolio, with particular emphasis on its oncology offerings, the company did discontinue development of a cancer candidate. The BTK degrader ABBV-101 was being studied in a phase 1 trial of 135 patients with relapsed or refractory non-Hodgkin’s lymphomas.

The study, which was designed to primarily assess adverse events and changes in certain lab results, was expected to read out in 2031, according to ClinicalTrials.gov.

“The decision is not an outcome of M23-647 clinical trial results,” an AbbVie spokesperson told BioSpace. “We remain committed to advancing research across blood cancers including B-cell malignancies to continue elevating the standard of care.”

AbbVie has already set about filling the void, bringing in three new assets across its pipeline in the last quarter. Internally, the Big Pharma has started clinical testing for antibody-drug conjugate ABBV-438, an intravenous drug designed to treat relapsed/refractory multiple myeloma.

On the business development side, AbbVie in January licensed RemeGen’s PD-1/VEGF bispecific for $650 million cash, plus up to $4.95 billion biobucks. The ex-China rights for the phase 1 candidate give the company a spot in a heated race that already includes Bristol Myers Squibb, Merck and Pfizer.

Another new addition is an early-stage clinical candidate from Kestrel Therapeutics. AbbVie’s deal for the oral pan-KRAS inhibitor, announced Tuesday, includes the exclusive right to acquire the entire biotech if certain milestones tied to the solid tumor candidate are met. Overall, the deal could be worth up to $1.45 billion.

The dealmaking—which also includes AbbVie’s recent pain medicine pact with Haisco Pharmaceutical—comes at a cost. Through the first quarter of this year, acquired in-process research and development (IPR&D) and milestones costs reduced the company’s earnings by $0.41 per share. Despite the hit, AbbVie is still bullish on BD.

“We have been and continue to be very open to acquiring external innovation, with a major focus for us in immunology, neuroscience, oncology and obesity,” AbbVie CEO Robert Michael said on the Wednesday earnings call.

“[If] we see a differentiated asset in any of these areas, whether early-stage, late-stage or even on market, we are very willing to pursue it,” Michael continued. The company’s current market portfolio and pipeline provide “a clear line of sight to very strong growth into the 2030s,” allowing AbbVie to operate from a position of “ample financial capacity,” according to the CEO.

While Michael views potential M&A as an opportunity to drive growth, he said it’s not required for AbbVie.

“While we don’t need BD to deliver top tier growth this decade, we’re not opposed to near-term revenue drivers that are differentiated in our core areas of focus,” he added.

Biogen’s growth was expected to stay flat through the 2030s. A key acquisition and busy late-stage pipeline have relieved the pressure and cleared the way for some early-stage bets, CEO Chris Viehbacher said Wednesday.

With a late-stage pipeline set to deliver in the 2030 timeframe and new revenue expected from the acquisition of Apellis, Biogen will now look for early-stage assets to fill a pipeline that CEO Chris Viehbacher admitted is “quite thin.”

“We’re looking at really building the next generation of growth,” the CEO told analysts on the company’s first quarter earnings call on Wednesday.

Viehbacher has previously said that deals for early-stage assets were the goal, but analysts were looking for the company’s renewed strategy after the acquisition of kidney disease biotech Apellis last month, which fell firmly outside this goal.

After that deal, Viehbacher said his business development team would be “more opportunistic” in terms of M&A specifically.

“I don’t think there’s any particular need at [this] point to do that, but, you know, I guess we’ll keep an eye out, but we’re not going to be doing the search,” he said.

This contrasts with the company’s strategy over the past two to three years, when the team has been actively searching for M&A opportunities.

“We have had a very systematic search and felt that we wanted to do M&A. I would say, with the Apellis transaction and with the pipeline, we don’t see the same need to be as proactive on that front.”

Consensus estimates had Biogen’s growth staying relatively flat through the 2030s. In an effort to bend that curve upward, Biogen has built a major late-stage pipeline anchored in lupus, nephrology and neurology. Then last month, the company offered $5.6 billion to buy Apellis.

“If we execute well . . . and the pipeline does come through this, this should be a company that can grow well into the into the 2030s and what we need to really be thinking about is what comes after that,” Viehbacher said.

Now is the time to look at some earlier-stage research bets that could pay off further down the line, Viehbacher said.

“Most of what we’re going to be focusing on is early-stage development and research, because that’s that part of the pipeline is quite thin,” he said. In particular, the CEO pointed to research stage projects, pre-investigational new drug application or Phase 1 assets.

With that said, Biogen has bolstered the early pipeline with several licensing collaborations, particularly in immunology with Vanqua Bio and Dayra Therapeutics. Viehbacher also pointed to the $46 million deal with City Therapeutics inked last year to work on RNAi-based therapies for central nervous system diseases.

Taking a beat

Otherwise, Biogen’s earnings split analysts with revenues outpacing consensus estimates by 10% at $2.5 billion, a 2% year-over-year increase. Mizuho’s researchers called it a “solid beat,” while Truist Securities dubbed the quarter a “low quality” beat.

Revenue was led by a 12% increase across Biogen’s growth products, including Alzheimer’s disease therapy Leqembi, which rose 74% year-over-year to $168 million in sales globally, and Skyclarys, which took home $151 million for 22% growth. Sales of postpartum depression drug Zurzuvae, meanwhile, doubled to $55 million.

“Leqembi sales are starting to show real momentum, beating consensus estimates by +12% as barriers to Alzheimer’s therapy decline and patient awareness are slowly increasing,” BMO Capital Markets noted.

Biogen also enjoyed “continued durability” of its multiple sclerosis franchise, which has been in decline for several quarters as multiple products face loss of exclusivity, according to Truist. The firm was particularly pleased by sales of Tysabri, which took home $441 million worldwide in the quarter after earning just $381 million for all of 2025.

Altogether, the franchiser took home $958 million, which was flat from the year before but a beat nonetheless, William Blair commented. Analysts had been expecting $841 million instead.

“A lot of the work that we have done over the last two, three years, is now coming to fruition,” Viehbacher said. “Now we are very conscious of the fact that in business, 10% is strategy, 90% is execution, and so we need to now execute as a team.”

The FDA has renewed calls for Amgen’s Tavneos to be pulled from the market, saying it has discovered new evidence that study personnel doctored the results of the drug’s pivotal study in order to make it look effective.

The FDA is stepping up its campaign calling for Amgen to remove its autoimmune therapy Tavneos from the market, casting doubt on the integrity of the evidence that led to the drug’s approval nearly five years ago.

“New information that only became known to CDER [Center for Drugs Evaluation and Research] more than three years after approval shows that unblinded study personnel manipulated the results of the pivotal clinical study so the drug looked effective when the original analysis did not support that conclusion,” the FDA said in a statement on Monday.

The regulator did not provide evidence to back up these allegations. The FDA added that Amgen violated the agency’s regulations by not disclosing the original analysis in its application.

CDER “can no longer conclude that there is, or has ever been, a valid demonstration that Tavneos is effective,” according to the press announcement.

“Patient safety guides every decision we make,” an Amgen spokesperson told BioSpace in an email. “We remain confident in TAVNEOS as a safe and effective medicine, supported by years of clinical data and real-world evidence. Our perspective on the benefit-risk profile of TAVNEOS differs from the Agency’s. We will evaluate next steps and respond to the FDA, while keeping patient needs and support at the forefront.”

Tavneos, an oral complement blocker, was approved in October 2021 for severe active anti-neutrophil cytoplasmic autoantibody-associated vasculitis. This condition, more commonly known as ANCA vasculitis, is a group of rare autoimmune inflammatory diseases that damage blood vessels. Tavneos was originally developed by ChemoCentryx, which Amgen acquired in 2022 for $3.7 billion.

The FDA first called for Tavneos’ withdrawal in January after the agency found problems with the way ChemoCentryx re-adjudicated the primary endpoint in the pivotal study to support the drug’s application. Amgen refused.

Then in March, the regulator put out a safety alert flagging 76 cases of liver injury and eight deaths in patients who were given Tavneos. These safety signals, which were detected through post-marketing surveillance, had “reasonable evidence of a causal association” with the drug, the agency said.

While Amgen has identified liver toxicity as a safety risk associated with Tavneos in its clinical program—a risk that is reflected in the product’s label—the cases outlined in the FDA’s alert, especially those with fatal outcomes, “represent new safety concerns,” the FDA said in its alert.

The FDA again pointed to Tavneos’ side effects in its statement on Monday, noting that CDER “is increasingly concerned about the safety profile” of the drug.

Despite ramping up its rhetoric against Tavneos, the FDA has not yet begun pulling levers to force Amgen to pull the product from the market. For now, Tavneos will remain available to patients and prescribers.