The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given Sobi’s Gamifant a final negative opinion after re-examining its initial decision.
Sobi requested that the CHMP re-examine the application for Gamifant (emapalumab) as a treatment for of primary haemophagocytic lymphohistiocytosis (HLH) after the Committee initially rejected the drug in July.
Primary HLH is a genetic disease characterised by widespread destruction of blood cells, extremely high iron levels in the blood, coagulation problems and excessive growth of organs.
The condition can be passed on genetically by parents who are carriers of the disease, or can occur as a spontaneous mutation. It results in an over-expression of IFN gamma that causes an auto-immune-like syndrome.
Patients with primary HLH are limited to haematopoietic stem cell transplantation (HSCT), a procedure which requires individuals to undergo intense treatment first in order for it to be successful.
In its original decision, the CHMP ruled that the results of the study used to support Sobi’s application for Gamifant were not convincing enough to conclude that the drug is effective in the treatment of primary HLH.
In addition, the CHMP said that the study only involved a small number of patients, who were also receiving other medicines used to treat HLH. This, the Committee said, made it difficult to determine if the responses seen in some patients were due to Gamifant treatment.
The CHMP also called into question the data concerning the safety of Gamifant, saying in a statement that the design of the study made it difficult to collect data on the drug’s side effects.
After re-examining the available data, as well as additional advice from a group of experts, the CHMP determined that although Sobi had addressed concerns over Gamifant’s safety profile, the other concerns still remained.
“This recommendation by the CHMP is disappointing given the significant unmet medical need which exists for patients with pHLH who have no approved therapies in Europe,” said Ravi Rao, head of R&D and chief medical officer at Sobi
“During the re-examination we worked extensively with physicians and patients and were able to resolve some but not all of the concerns raised by EMA.
“We are confident about the clinical profile of emapalumab and our focus is now on increasing access for patients in other regions and developing new indications for this medicine,” he added.
Sobi, based in Stockholm, Sweden, acquired the global marketing rights to Gamifant from Novimmune in July 2018, and in June 2019 the company spent $518m to acquire the outstanding intellectual property and patents for the drug.
Gamifant was approved by the US Food and Drug Administration (FDA) for the treatment of primary HLH in November 2018. In the US, over 100 primary HLH patients have been treated with Gamifant, with the benefit/risk profile continuing to prove ‘favourable’ according to Sobi.