European CHMP Delivers Negative Opinion of Gamifant; Sobi Requests Re-examination

European CHMP Delivers Negative Opinion of Gamifant; Sobi Requests Re-examination

The Committee for Medicinal Products for Human Use (CHMP) recommended denying Sobi‘s marketing authorization request for Gamifant (emapalumab) for the treatment of primary hemophagocytic lymphohistiocytosis (HLH) in children in Europe younger than 18 years.

Sobi stated in a press release  it will ask the CHMP to re-examine the request, as Gamifant would address a significant unmet need among primary HLH patients given that Europe currently has no approved treatment for this condition. Sobi expects an opinion by the end of the year.

The U.S. Food and Drug Administration (FDA) approved Gamifant in 2018, making it the first approved therapy for primary HLH in the U.S. According to Sobi, more than 100 patients have been treated in the U.S. to date and the results have been favorable so far.

“Emapalumab has demonstrated a positive benefit/risk profile in primary HLH in a post-approval real life setting in the US since the FDA approval in 2018. The product has been able to make a substantial difference for a very vulnerable group of patients in the US,” said Guido Oelkers, CEO and president of Sobi.

Primary HLH is characterized by an overactive immune system that causes excessive inflammation. Often appearing in infancy, it is associated with high morbidity and mortality.

Gamifant is an antibody that targets interferon (IFN)-gamma, a molecule that plays a key role in the inflammatory response by activating other immune cells.

“In my role as principal investigator of the NI-0501-04/05 studies in Europe, I was significantly surprised about the EMA decision not to approve emapalumab for children with primary HLH who failed or are intolerant to front-line therapy,” said Franco Locatelli, MD, PhD, the principal investigator in the European studies.

“I had the privilege to observe that this monoclonal antibody … was well tolerated and effective in a large proportion of the patients, representing a model of precision medicine. While U.S. children have since almost 2 years the possibility to be treated with this novel, safe, highly effective and targeted therapy, the EMA decision paves the way for migratory health flows towards non-European Centers that can grant this treatment,” Locatelli said.

Michael Jordan, MD, principal investigator in the U.S. studies confirmed Locatelli’s opinion: “The NI-0501-04/05 studies have demonstrated that emapalumab has clear therapeutic activity in primary HLH and have validated interferon gamma as a key target in these patients,” Jordan said. “I am grateful for the opportunity to help lead these trials which were conducted with the greatest rigor and transparency … I believe that emapalumab will benefit patients around the world with HLH.”

According to Oelkers, Sobi now will “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.”

Of note, positive results from a study evaluating Gamifant’s safety and effectiveness in patients with primary HLH were published recently in the New England Journal of Medicine.

Sobi also has plans to test Gamifant’s usefulness as a preemptive treatment among patients with risk factors for acute graft failure hematopoietic stem cell transplant, a potentially life-threatening condition. The company believes that Gamifant has the potential to be used in other conditions affected by IFN-gamma activity, expanding its market potential.

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