Meet Baby Dennis. He suffers from spinal muscular atrophy, a disease that robs people of physical strength by affecting the motor nerve cells in the spinal cord, taking away the ability to walk, eat or breathe.
According to Israel Hayom, there is only one known cure for the disease: ZOLGENSMA, a gene therapy that must be administered to children before the age of 2 to be effective. His mother, Nadia Bieber of Kiryat Arba, has been trying to get her son this life-saving treatment. But she told Israel Hayom that because the drug costs NIS 7.6 million per treatment and is not included in the services basket, she has been unable to access it - and her son is running out of time.
In Israel, there are about 130 patients of all ages with SMA muscular dystrophy. According to estimates provided by the Hebrew daily, there are 15 to 20 two-year-old children with the disease.
ZOLGENSMA is manufactured by AveXis, a clinical-stage gene therapy company, which was acquired by Novartis in mid-2018.
According to Israel Hayom, the drug was posed to be discussed by the drug committee in the fall. The discussion will likely result in a public battle due to its cost. Either way, by then it will be too late for Dennis.
Bieber appealed to the Meuhedet health fund of which she and her son are a part, for an exception. Meuhedet refused to honor her request.
This is partly because now there is another drug that was approved by the Healthy Ministry last year that works for the same disease: Spinraza. But this drug must be given regularly throughout the patient's life.
Approximately 110 patients have been treated in this country, with the drug being given three times a year indefinitely, and according to information from doctors and parents of children treated with the drug, it has already produced very good results, reported the Hebrew daily.
Dennis is already receiving this drug, but his doctor felt he “meets the exact criteria” for ZOLGENSMA and that it could improve and even save his life before it is too late. Dennis was diagnosed with the disease at the age of one and a half, and he is already suffering from severe motor retardation along with normal mental development.
Although a lawyer who worked on behalf of Bieber demonstrated to Meuhedet that the new drug will work much better for Dennis than the one he currently receives, and that over the course of his lifetime, the one-time administration of ZOLGENSMA would cost less than the continual administration of Spinraza, the health fund still refused to make this exception.
“The fund's reasons for refusal also do not meet the standards of mandatory court rulings and the Ministry of Health's directive,” Bieber’s lawyer told Israel Hayom.
The health fund did not return the paper’s requests for comment.Last month, a campaign on a Jewish crowdfunding website raised more than $2 million in five days to provide this same drug to an almost 2-year-old girl in Florida. In America, the one-time dose of ZOLGENSMA cost $2.125 million. It is covered by insurance.
The crowdfunding campaign on The Chesed Fund website raised $2,202,364.80 from 23,353 donors between July 12 and July 17. Eliana Cohen received her treatment a day before her 2-year birthday.