CHMP Rejects SOBI’s HLH Therapy in Kids 18 and Under, Company Seeks Reexamination

Rare Daily Staff

Swedish Orphan Biovitrum said that the Committee for Medicinal Products for Human Use is recommending a refusal of the marketing authorization for emapalumab for the treatment of primary hemophagocytic lymphohistiocytosis in children under 18 years of age in Europe.

Primary hemophagocytic lymphohistiocytosis (HLH) is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. Despite some treatment advances, there continues to be a high unmet medical need, in particular, in patients that have failed conventional therapy as there are no approved treatment options outside the United States.

Emapalumab is a monoclonal antibody that binds to and neutralizes interferon gamma. In the United States, emapalumab is indicated for pediatric and adult primary HLH patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy.

In the United States, emapalumab is the first therapy approved by the U.S. Food and Drug Administration for primary HLH. More than 100 patients have been treated in the United States and the company said the benefit/risk profile continues to be favorable.

Sobi said it will request a re-examination by the CHMP with an expected opinion by end of year 2020 because of the significant unmet medical need that emapalumab addresses in patients with primary HLH who have no approved treatments in Europe.

“The product has been able to make a substantial difference for a very vulnerable group of patients in the U.S. During the last years, our team has gained a lot of experience in this rather complex disease area,” said Guido Oelkers, CEO and president of Sobi. “We will do our utmost to share these insights and address the open questions by CHMP during the re-examination with a view to secure access for primary HLH in children to this treatment in Europe.”

Photo: Guido Oelkers, CEO and president of Sobi