As Amgen prepares to defend Tavneos’ market approval in the U.S., the company has received a bad omen overseas, with regulators recommending that the ANCA-associated vasculitis drug lose its authorization in the European Union after a new review led to the conclusion that its benefits are “no longer proven to outweigh its risks.”
That was the take from the European Medicines Agency, which issued a roundup of recent approval decisions—both positive and negative—from its Committee for Medicinal Products for Human Use (CHMP) on Friday. Once the CHMP has delivered marketing authorization recommendations, final sign-off on the decisions must still be conferred by the European Commission.
In the case of Tavneos—originally approved in two rare forms of ANCA-associated vasculitis in the EU in 2022—the CHMP has now wrapped up a review of the drug that started earlier this year over data integrity questions from the main study that supported its approval.
At the time of its original authorization, and based on data from the 331-patient Advocate study, European regulators had determined that Tavneos was “at least as effective” as a 20-week course of high-dose corticosteroids at inducing remission in patients with active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).
But the CHMP has now determined that the Advocate study was “conducted in breach of good clinical practice (GCP) principles,” adding that the data provided for the assessment “were found to be incorrect and misleading and could no longer be relied upon for demonstrating Tavneos’ effectiveness.”
Moreover, post-marketing data and other post-hoc analyses “are not considered sufficient” to argue in favor of the drug’s benefits, per the CHMP.
A final decision is now expected by the European Commission “shortly,” Vifor Fresenius Medical Care Renal Pharma and CSL—which market Tavneos in Europe—said in a June 26 release.
"While we are disappointed in the outcome of the Article 20 procedure, we will respect the outcome of the regulatory process and are committed to implementing it in full, Bill Mezzanotte, head of R&D at CSL, said in a statement.
No new patients will be treated with Tavneos in the EU, and patients currently on the drug should talk their physician about next steps, CSL added.
Stateside, Amgen is gearing up for an FDA hearing to address the regulator’s efficacy and safety concerns over Tavneos, recently winning a one-month extension on a data deadline, which is now set for July 29, according to Bloomberg News.
Earlier this year, Amgen refused an FDA request to stop selling Tavneos in the U.S., with the agency stepping up its efforts to potentially curtail its use in late April when it formally proposed to withdraw the medication.
"We are deeply concerned about the potential impact that the CHMP recommendation will have on patients and healthcare providers who value access to Tavneos as a treatment option," a spokesperson for Amgen told Fierce in an emailed statement, reaffirming that CSL is leading all EMA interactions on the drug across the pond.
"Amgen continues to believe that Tavneos is an important treatment option for people living with AAV with a favorable benefit-risk profile based on all available data including over 20 real-world studies," the spokesperson added. "The deadline for our hearing submission has been extended to July 29, and we remain engaged with the FDA as the regulatory process continues in the United States."
A busy week for CHMP
Tavneos isn’t the only drug to get the cold shoulder from European regulators this week.
In particular, MaaT Pharma said Friday that it will seek a reexamination of its application after the CHMP adopted a negative opinion on conditional marketing authorization for its acute Graft-versus-Host disease (aGvHD) prospect, MaaT013.
As it stands, the CHMP believes that given the use of concomitant therapies to manage aGvHD, MaaT’s data package “does not allow sufficient attribution of the observed clinical effect and safety to the study treatment alone,” the company said in a Friday release.
Under EMA procedures, MaaT expects to receive its new opinion within 60 days of its reexamination request potentially being approved, which could set up a new verdict sometime around the middle of September, per the company.
MaaT is developing what it calls microbiome ecosystem therapies to help “restore balance and maximize clinical benefits for patients with severe, treatment-induced dysbiosis in acute diseases.”
Aside from MaaT’s drug, which the company hopes to market as Xervyteg, the CHMP also handed out negative opinions to the Netherlands Cancer Institute’s Tacquell in advanced melanoma and Yartemlea, from Omeros, to treat adults and kids ages two and older with hematopoietic stem cell transplant (HSCT)-associated thrombotic microangiopathy
On the other end of the spectrum, the CHMP also doled out several positive recommendations to big-name drugs this week, including Eli Lilly’s BTK inhibitor Jaypirca, which has received the regulator’s blessing to treat adults with chronic lymphocytic leukemia (CLL) across all lines of therapy, regardless of prior BTK inhibitor treatment. The European Commission is expected to make a final determination “in the next one to two months,” according to Lilly.
The positive recommendation, fueled by results from the BRUIN CLL-313 and -314 studies, bodes well for the drug’s chances in the indication in the U.S., where the FDA is expected to weigh in on approval in the second half of 2026.
Meanwhile, Incyte’s topical JAK inhibitor is inching closer to its second EU indication after the CHMP put its weight behind the cream-based treatment’s approvability in adults with moderate atopic dermatitis. If given EC sign off, Opzelura would become the first steroid-free, topical JAK treatment available for this patient population in Europe, Incyte noted Friday.
The CHMP based its decision on data from the phase 3 TRuE-AD4 study, plus supporting data from the separate late-stage trials TRuE-AD1 and TRuE-AD2, with the first, pivotal study showing that Incyte’s product significantly improved clinical signs and symptoms of moderate atopic dermatitis, including itch, as early as day 2 of treatment.
Opzelura was first approved in the U.S. as a treatment for atopic dermatitis in 2021 before scoring a green light in vitiligo the following year. Over in Europe, it currently holds one approval to treat non-segmental vitiligo with facial involvement in adults and adolescents ages 12 years and older.
Aside from a clutch of other potential label extensions in the EU, the CHMP further granted recommendations to the following new drugs: The flu vaccine Aujemflu; the Parkinson’s disease with motor fluctuations treatment Hopledo; and Onswik to treat type 2 diabetes in adults.
And, following a reexamination of the sort MaaT is now seeking, Acadia Pharmaceuticals reversed the negative opinion it had previously received from the CHMP on its Rett syndrome treatment Daybu.
The regulator also gave its blessing to a pair of biosimilars referencing Prolia and Xgeva and Neulasta.
Editor's note: This story has been updated with a comment from Amgen, and to clarify in the headline that Amgen does not control rights to Tavneos in Europe.