Sobi has announced its intent to file for a re-examination of Gamifant (emapalumab) in Europe following negative opinion by the European Medicines Agency’s human medicines committee.
The Committee for Medicinal Products for Human use (CHMP) adopted a negative opinion recommending refusal of marketing authourisation for the drug as treatment of primary haemophagocytic lymphohistiocytosis (HLH) in children under 18 years of age in Europe.
‘Given the significant unmet medical need that emapalumab addresses in patients with primary HLH with no approved treatments in Europe, Sobi will be requesting a re-examination by the CHMP with an expected opinion by end of year 2020,’ the firm noted.
Primary HLH is a rare syndrome associated with high morbidity and mortality for which there continues to be high unmet medical need, in particular in patients that have failed conventional therapy as there are no approved treatment options outside the US, Sobi noted.
In the US, emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for the condition.
“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, Sobi’s president and chief executive.
“During the last years our team has gained a lot of experience in this rather complex disease area. 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”.
Sobi also plans to initiate clinical studies with emapalumab for potential indications such as pre-emptive treatment of patients with risk factors of HSCT acute graft failure, which will further expand the drug’s target patient population and market potential.
The company is expecting peak sales of more than $500 million, which remains unchanged regardless of an approval in Europe.