The American company Amylyx which developed a drug to treat the muscular dystrophy disease ALSwhich received marketing approval in the US recently, signed an agreement with the Israeli company Neopharm, to commercialize the drug in Israel. The agreement in Israel is the first signed by the company outside of North America.
ALS is a disease characterized by the loss of muscle control which gradually leads to an accumulation of disabilities and finally death when control of breathing is lost, on average about two to five years after diagnosis. The disease also affects young people, and received a lot of attention following the “Ice Bucket Challenge” and in Israel also following the activities of the Israels and Prize4life associations. About 700 people with ALS live in Israel. Slowing down the development of the disease is expected to increase the market.
Following the public attention, for the first time in the last two decades the strenuous work of scientists from all over the world began to decipher the mechanism of the disease. Today it is impossible to say that the mechanism of the disease has been deciphered, but there is significant progress, after genes that increase the prevalence of the disease and biological markers that indicate its existence have been found.
Amylyx’s drug does not cure ALS, but only slightly slows down the course of the disease, and that too according to a relatively small trial. In March 2022, an advisory committee of the FDA (American Food and Drug Administration) decided not to recommend the drug’s approval, with a majority of 6 opposed to 4 in favor. However, even those who voted against the drug in the same vote said that the decision was very difficult and borderline.
Amylyx insisted and submitted to the FDA another analysis of the results of the trial, in September 2022 another meeting of the advisory committee was held, in which the creators reversed – 7 in favor and 2 against. Following this, the FDA approved the marketing of the drug, with the possibility of changing the decision based on a larger trial that the company is conducting these days, which should end in 2024.
In the US, the stated cost of the drug is $158,000 per year, a price that has been criticized due to the drug’s modest clinical effectiveness. But the company is already working with patient organizations and insurance companies to obtain discounts and support for patients who cannot afford to pay.
Neopharma stated that the approval of a drug already approved in the US takes an average of 12-18 months. This length of time for the approval of a drug already approved by the FDA, and all its considerations and data that guided it are visible to the Israeli regulator, is considered long and many in the Israeli pharmaceutical world visit the office Health on this, when the ministry also has its arguments that it does not want to be a ‘rubber stamp’ of the FDA, and on the other hand it does not have a budget to speed up the process. It is possible that in this case, the approval will be faster, because 18 flaps may be the life expectancy of a sick person in this disease.
It is not yet known what the price of the drug may be in Israel.