Europe’s Committee for Medicinal Products for Human Use (CHMP) has given a thumbs-down (PDF) to Sanofi’s Rezurock as a third-line treatment for chronic graft-versus-host disease (cGVHD).
The European Medicines Agency’s panel of experts issued its negative opinion because it “was difficult to quantify” the effect Rezurock had on patients relative to other treatments. Additionally, the CHMP noted that a study in which patients used Rezurock as a first-line treatment “did not show it had any beneficial effects.”
The CHMP also stated that due to the need for new treatments in this indication, it considered recommending Rezurock for a conditional marking nod. But the regulator ultimately decided not to, given that Sanofi isn't in a position to provide more efficacy data on the drug until 2030.
The company plans to seek a reexamination of the opinion.
“Sanofi is confident in the body of clinical and real-world evidence that underscores Rezurock’s consistent efficacy and well-established safety profile,” Olivier Charmeil, Sanofi’s executive vice president of general medicines, said in a release. “We will continue to work closely with the European Medicines Agency with the aim of bringing this treatment to patients in the EU who are waiting.”
Out of 93 recommendations this year on new medicines, the Rezurock snub represents the sixth negative opinion from the CHMP so far.
Sanofi gained Rezurock in a $1.9 billion buyout of Kadmon in 2021, less than two months after the FDA approved the treatment for third-line use in cGVHD. Today, the medicine is available in 20 countries, including the U.K., Canada and China.
More than 17,000 patients have been prescribed Rezurock since its initial U.S. nod, Sanofi said Friday. Sales of the treatment reached 132 million euros ($154 million) in the first half of this year, which was a 21% increase over the same period in 2024.
A whirlwind of endorsements
It wasn't all bad news for Sanofi this week, however, as the CHMP recommended the company’s Bruton tyrosine kinase (BTK) inhibitor Wayrilz for approval to treat immune thrombocytopenia in adult patients who are refractory to other therapies.
The positive opinion comes after the FDA approved the potential blockbuster in August. Sanofi gained the drug—along with another BTK hopeful tolebrutinib—in 2020 through its $3.7 billion acquisition of Principia Biopharma.
The CHMP also signed off on Insmed’s first-in-class treatment Brinsupri for non-cystic fibrosis bronchiectasis. The recommendation comes two months after the FDA endorsed the dipeptidyl peptidase 1 (DPP1) inhibitor. The oral treatment is indicated for patients ages 12 and older who have had at least two flare-ups of the lung disorder over the previous year.
Apart from the new drug endorsements, the CHMP issued further positive opinions this week, which would expand the label for eight previously approved drugs, including Pfizer’s COVID antiviral Paxlovid, Johnson & Johnson’s autoimmune treatment Tremfya, Regeneron’s skin and lung cancer therapy Libtayo, Agios’ anemia drug Pyrukynd and CStone’s lung cancer medicine Cejemly.
Also making the grade were the following blood cancer drugs: Bristol Myers Squibb’s Breyanzi, Roche’s Gazyvaro and Novartis’ Scemblix.