US FDA approves first HLH treatment in 24 years

By Maggie Lynch

- Last updated on GMT

(Image: Getty/AndreyPopov)
(Image: Getty/AndreyPopov)

Related tags: Fda, Sobi, Gamifant

Sobi’s Gamifant is approved as a treatment for pediatric and adult patients with primary hemophagocytic lymphohistiocytosis.

Gamifant (emapalumab-lzsg) is an interferon gamma (IFNy) blocking monoclonal antibody (mAb), approved for patients whose hemophagocytic lymphohistiocytosis (HLH) is refractory, recurrent or progressive or have an intolerance to conventional therapy.

Gamifant was provided priority review and had received an orphan drug designation, breakthrough therapy designation, and rare pediatric disease designation by the US Food and Drug Administration (FDA).

The drug is administered concomitantly with dexamethasone, with 63% of patients displaying an overall response to treatment in clinical trials.

Development and licensing

Gamifant is the first drug specifically targeted to neutralize IFNy, said Cristina de Min, chief medical officer at Novimmune. She further stated that additional clinical studies are ongoing or planned with the drug for diseases in which IFNy is considered pathogenic.

Primary HLH is an ultra-rare syndrome, with a high morbidity and mortality. Previous to the FDA decision, no treatments specifically for the syndrome had been approved.

Additionally, Michael Jordan, a physician-scientist in the division of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's Hospital Medical Center HLH Center of Excellence, said in a statement, "Emapalumab represents an entirely new approach to treating primary HLH and helping these very sick patients reach hematopoietic stem cell transplant."

Gamifant was developed and submitted for approval to the FDA by Novimmune. Sobi acquired the global rights to Gamifant from Novimmune through a licensing agreement that was previously announced in July 2018.

Rami Levin, president of Sobi North America told us, that the company made an upfront payment to Novimmune of $50.2m (€44m) in cash for the global license of the drug. A total of $401.9m in additional payments will be made over an eight-year period.

Gamifant is expected to be available for administration in treatment centers in the US in Q1 of 2019.

Related topics: Markets & Regulations, Pipelines

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