BioPharma, Pharma

FDA approves Sobi’s Gamifant for rare immune disease

The Swedish drugmaker acquired rights to the drug from Switzerland-based Novimmune in July for about $50 million, plus about $400 million in payments over eight years.

Text FDA Approved appearing behind ripped brown paper.

A Swedish drugmaker has won Food and Drug Administration approval for a rare disease drug that it bought only months ago.

The FDA approved Stockholm-based Sobi’s Gamifant (emapalumab), for primary hemophagocytic lymphohistiocytosis, or HLH, in children and adults. The approval was based on a clinical trial of 27 children with suspected or confirmed primary HLH whose disease was refractory, recurrent or had progressed on conventional therapy. Of the patients, 63 responded to Gamifant treatment, and 70 percent were able to receive stem cell transplant. Sobi anticipates the drug has peak sales potential of 2.5-3 billion Swedish kronor, or $276.8-$332.2 million.

HLH is an immune-activation disease that affects around 5,000 people in the US, European Union and Japan, mainly infants and young children, according to Sobi. Before Gamifant’s approval, the disease – which is life-threatening if untreated – had no approved therapy and was mainly treated with chemotherapy drugs to suppress immune response. The disease causes the body’s immune cells to damage organs like the liver, brain and bone marrow.

Sobi bought global rights to the drug in July from privately held Swiss drugmaker Novimmune. Financial terms of the deal included an upfront payment of 50 million Swiss francs – $50.3 million – and 400 million francs – $402.5 million – in payments over an eight-year period, though both companies have the option to accelerate the payments any time after July 1, 2019.

The news comes less than a week after Sobi bought US rights to the AstraZeneca drug Synagis (palivizumab), currently the only drug approved as a prophylactic treatment for respiratory synctial virus in high-risk infants, as well as the right to receive 50 percent of US earnings from MEDI8897, an investigational AstraZeneca drug also for RSV. Sobi paid 13.6 billion kronor, or $1.5 billion, for the drug, which was approved in 1998 and had global sales of $687 million worldwide, including $317 million in the US. Sales in the us had declined by 2 percent due to American Academy of Pediatrics guidelines that restricted its availability, while sales elsewhere in the world – where AbbVie is responsible for commercialization in more than 80 countries – increased by 5 percent, to $370 million.

Photo: Michail_Petrov-96, Getty Images