In a flurry of decisions Friday, the European Medicines Agency’s human products committee recommended 17 new drugs for approval while letting Takeda withdraw from market an inflammatory bowel disease complication medication.
Merck & Co.’s kidney cancer drug Welireg, Bayer and BridgeBio’s transthyretin amyloid cardiomyopathy (ATTR-CM) therapy acoramidis, and Gilead’s primary biliary cholangitis (PBC) treatment seladelpar were among the 17 for which the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued positive opinions.
For Welireg in third-line clear cell renal cell carcinoma and in tumors associated with von Hippel-Lindau disease, the CHMP is recommending a conditional marketing authorization. Both indications have counterpart approvals from the U.S. FDA.
The CHMP based its positive opinion on tumor shrinkage data from the phase 2 LITESPARK-004 trial and the phase 3 LITESPARK-005 study, as well as Welireg’s progression-free survival win against Novartis’ Afinitor in the latter study. However, a final analysis of the -005 trial didn’t spot a statistically significant overall survival benefit for the Merck drug in clear cell kidney cancer despite a favorable trend.
Analysts have previously figured that Welireg, which Merck got from a 2019 acquisition, holds blockbuster potential if it can reach earlier kidney cancer settings.
Moving to acoramidis, which will bear the brand name Beyonttra once the European Commission adopts a formal marketing authorization. Bayer obtained European rights to the ATTR drug earlier this year by paying BridgeBio $310 million in upfront and near-term milestones. Both stabilizers of transthyretin, acoramidis is a direct competitor to Pfizer’s blockbuster tafamidis franchise.
The CHMP endorsement comes after the FDA approved the drug under the commercial moniker Attruby late November. The drug’s U.S. label cites its benefit on the composite measurement of all-cause mortality and cardiovascular-related hospitalizations but not death reduction by itself.
For its part, the CHMP in its summary also cited Beyonttra’s improvement on the composite endpoint. Detailed recommendations will be made available following the European Commission’s final decision, which Bayer said is expected in the coming months. Pending approval, Bayer said it plans to launch Beyonttra in Europe in the first half of 2025.
As to Gilead’s seladelpar, which gained the FDA’s go-ahead for the treatment of PBC in August, the CHMP reviewed the PPARδ agonist under the agency’s Priority Medicines pathway, which provides extra support for promising medicines with a potential to address unmet medical needs. Seladelpar came to Gilead via the Big Biotech’s $4.3 billion buyout of CymaBay Therapeutics earlier this year.
The CHMP’s endorsement of seladelpar, known as Livdelzi in the U.S., comes shortly after a court of the European Union backed the European Commission’s decision to revoke the PBC nod previously granted to Intercept and Advanz Pharma’s Ocaliva.
Speaking of market withdrawal, Takeda has decided to pull its Alofisel from the EU market as a treatment for complex anal fistulas in Crohn’s disease. The move comes more than a year after the stem cell therapy was found to be no better than placebo in a phase 3 trial dubbed ADMIRE-CD II. Additional results presented this February again showed no statistical differences in any of the secondary endpoints. Takeda agreed to the study because the original trial supporting the approval, ADMIRE-CD, was relatively small in size with modest benefits observed.
According to the EMA, Takeda is voluntarily pulling Alofisel as the company considered it impossible to provide additional data to meet the agency’s requirements.
Takeda had months of discussions with the EMA, healthcare professionals and other stakeholders on the totality of data around Alofisel, Ramona Sequeira, head of Takeda’s global portfolio division, said in a statement Friday.
“Those valuable discussions indicated that despite the conflicting results across our data, the clinical benefit of Alofisel is no longer sufficient to justify its continued use in the EU,” Sequeira said.
Takeda said it started reaching out to doctors and others about this development earlier today, and that it’s talking to other health authorities in countries where Alofisel is approved. About 1 in 3 Crohn’s patients would develop a fistula, which is marked by abnormal passages between the lower parts of the gut and the skin near the anus.
Alongside those decisions, the CHMP also considered Eli Lilly’s application to expand the label of Mounjaro to cover obstructive sleep apnea in obesity patients. But the drug reviewers figured that Mounjaro’s existing indication for weight management already covers this use and that it does not need a separate indication.
Among drugs that earned label expansions from the CHMP, Lilly’s Omvoh, a direct competitor to AbbVie’s blockbuster Skyrizi, won a recommendation for use in Crohn’s disease.
Boehringer Ingelheim’s Ofev has become the first med recommended for treating progressive fibrosing interstitial lung disease in children from the age of 6. The condition is rare, affecting less than 5 children per million in the EU. The drug will come in a lower strength for children than for adults.
GSK’s PD-1 inhibitor Jemperli, used in combination with chemotherapy, won an extension to treat all first-line patients with primary advanced or recurrent endometrial cancer. The indication was previously limited to mismatch repair deficient or microsatellite instability-high tumors.