Sobi snags FDA nod for rare disease drug Gamifant

The FDA approved Sobi's Gamifant to treat certain patients with primary HLH. (FDA)

Several months after scooping up global rights to emapalumab in a $452 million deal, Sobi has won approval for the drug to treat certain patients with primary hemophagocytic lymphohistiocytosis. The FDA nod marks the first treatment improvement for the patient group in decades, and Sobi will market the new drug as Gamifant.

The rare disease drugmaker plants to launch Gamifant in early 2019 and didn’t immediately disclose pricing. It picked up rights to the drug in a July deal worth up to $452 million with Swiss biotech Novimmune, which developed the drug.

At the time, Sobi said it believed Gamifant could generate peak sales of up to $330 million. The rights deal comprised about $50 million up front, and up to $400 million in milestones.

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Primary HLH is an ultrarare disease in which patients’ overexpression of interferon gamma is thought to cause the immune system to attack a patient’s own body, according to Sobi. Symptoms typically develop within the first year of life and the disease can be fatal if untreated.

RELATED: AstraZeneca offloads RSV drug to Sobi for $1.5B, its largest divestiture in 5 years

Gamifant won FDA approval to treat adult and pediatric primary HLH patients who don't respond to conventional HLH therapy. It’s now the only FDA-approved primary HLH med, according to the company.

Rami Levin, Sobi’s president of North America, previously told FiercePharma by email that the company “will leverage our existing North America sales force to educate" physicians about primary HLH and the drug.

“This team is already active in the field, and they have experience increasing awareness with physicians about rare diseases,” Levin added.

The Gamifant approval follows a separate large rights deal for Sobi with AstraZeneca. Last week, AZ announced it would be offloading RSV drug Syangis to Sobi for $1.5 billion upfront and a total deal value of $2.3 billion. More than 100 AZ employees are joining Sobi under the agreement.


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