On Oct. 15, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) confirmed its recommendation to refuse EU marketing authorization for Yartemlea, citing unresolved questions about supporting evidence.
Yartemlea is EMA’s listed name for narsoplimab. Omeros sought authorization to treat adults and children two years and older with thrombotic microangiopathy, a serious complication of stem cell transplants. The committee first issued a negative opinion on June 25; the October opinion followed a re-examination.
EMA said the adult evidence rested on a one-group study of 28 people who received the medicine without a placebo or another treatment for comparison. The agency concluded that methodological concerns meant the study could not establish the medicine’s effectiveness. It also said the pediatric evidence was too limited to support the proposed dose or assess safety and effectiveness in children.
Omeros said an eight-member expert group of hematologists, statisticians and transplant physicians took part in the re-examination, along with two patient representatives. The company said CHMP minutes recorded the group’s recommendation for external experts to review patient-level data. Omeros also said it submitted patient-level data in August and CHMP did not provide those data to the group.
Omeros said it planned to end the EU compassionate-use program, citing rising demand in the United States. The agency said people currently receiving the medicine through the program would have enough supply to finish their treatment courses.
