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World’s priciest drug used for first time on 2-year-old Israeli patient

“The effect of the medication will be seen in the coming months… we are full of hope,” said the pediatric intensive care director. 

By World Israel News Staff 

Zolgensma, said to be the most expensive medication in the world, has been used for the first time in Israel.

It was administered on Thursday to Liam Naji of Petah Tikva, a two-year-old with spinal muscular atrophy (SMA) who is being treated at Dana-Dwek Children’s Hospital located in Ichilov Medical Center in Tel Aviv, according to an Ichilov statement.

SMA is a “group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting,” according to the U.S. Genetic and Rare Diseases Information Center.

“The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood,” says the center.

In the U.S., the Food and Drug Administration approved the gene therapy but there has been “outrage over the drug’s price: $2.125 million,” according to Nathan Yates, 30, who has lived with the condition his entire life and authored a blog article published in May in STAT, an American-based medical news outlet.

The medication was administered to the little boy at the Israeli hospital through a vein transfusion. It was carried out “successfully and without complications,” according to Dr. Efraim Sdot, director of the pediatric intensive care unit.

“The effect of the medication will be seen in the coming months,” he said, adding that “we are full of hope and believe that Liam will be capable of growing up as a healthy and independent child.”

The medication can only be administered up to the age of two so that it’s not applicable for Yates. However, he defends the cost of the drug.

“Sure, it’s the world’s priciest drug,” Yates writes in STAT. “But instead of debating the level of financial profit that is appropriate for Novartis [Swiss-based pharmaceutical company], let’s focus on the needs of patients. How are we going to get treatments for rare diseases if there’s not a financial incentive for doing it?”

In the Ichilov statement, Liam’s parents thank the physicians who fought on behalf of their son and the Clalit HMO “which helped us in gaining the approval [of state funding] for the expensive medication.”