The approval for Gamifant is for paediatric, both new born and older, and adult HLH patients whose condition is refractory, recurrent or progressive or are intolerant to conventional HLH therapy.
Gamifant is supposed to be administered through intravenous (IV) infusion over one hour twice a week until haematopoietic stem cell transplant (HSCT).
The drug, which binds to and neutralizes interferon gamma (IFNy), was developed and submitted for approval to the FDA by Novimmune. Sobi purchased the global rights to Gamifant from the Swiss biopharma company through an exclusive licensing agreement signed in July 2018.
The monoclonal antibody is now the first and only drug to be approved in the US for the treatment of the hematologic disorder. Considered to be an ultra-rare syndrome of hyperinflammation, HLH normally occurs within the first year of life and can quickly turn out to be fatal unless diagnosed and treated.
FDA center for drug evaluation and research office of hematology and oncology products acting director Richard Pazdur said: “Primary HLH is a rare and life-threatening condition typically affecting children and this approval fills an unmet medical need for these patients.
“We are committed to continuing to expedite the development and review of therapies that offer meaningful treatment options for patients with rare conditions.”
Gamifant’s FDA approval was given based on the findings of a phase 2/3 study in 27 pediatric patients with primary HLH. The primary endpoint of the trial was met, with a clinically meaningful and statistically significant portion of patients showing an overall response at the end of treatment.
Apart from that, 70% of patients moved on to haematopoietic stem-cell transplantation (HSCT).
Sobi chief medical officer and research & development head Milan Zdravkovic said: “Primary HLH is a very rare disease with significant morbidity and mortality. The approval of Gamifant as the first treatment for primary HLH is a significant step in our journey of helping these patients.”
Earlier this month, Sobi agreed to acquire the US rights of AstraZeneca respiratory drug Synagis (palivizumab) in a deal worth $1.5bn.