Thanks to Amylyx Pharmaceuticals, Canada becomes the first country worldwide to have access to an ALS drug branded Albrioza (originally AMX0035). According to an article in Yahoo News, the drug was approved earlier this week. Canada now has three drugs to treat approximately three thousand ALS patients. The new drug, however, will not be available for several months as it was approved “with conditions.” According to regulations set out in Canadian law, the approval is conditioned upon the endpoints from a Phase III study involving six hundred patients.
The company, Amylyx, in cooperation with the Canadian health insurance system, must first negotiate pricing individually with thirteen provinces. This process may take six to nine months. But advocates remind patients that the normal process would have taken almost three years.
The decision is a decided win for Amylyx as it has used its resources on just one drug. Also, ALS is an extremely challenging disease.
A Three-Month PDUFA
The Prescription Drug User Fee Act (PDUFA) was passed by the US Congress in 1992 allowing the FDA to charge drug manufacturers fees to expedite the approval process. In the case of Amylyx, this establishes that a pivotal decision must be made by September.
The USFDA would like more time to review the new drug application and therefore pushed its decision to the latter part of September 2022. The FDA’s advisory committee, though, voted against Amylyx’s drug by 6 to 4, diminishing hopes for regulatory approval.
Amylyx Pharma hopes to have its data ready by 2024.
About the USFDA’s Drug Approval Process
The USFDA is well known for approving drugs before they are approved in other countries. But the opposite has been true for ALS drugs. As an example of patients desperate for help, Mitsubishi Tanabe Pharma received approval for edaravone, an ALS drug also approved in South Korea and Japan. Edaravone was not approved in the US until two years later.
Patients and their families who could afford it traveled overseas. They paid out of pocket for edaravone.
Due to the FDA’s delay, it is assumed that the same situation may arise with Albrioza while patients are waiting for the USFDA to complete its review. Or, it is possible that the situation may arise even if the drug is rejected by the FDA.
Both drugs – already approved in Canada – appear to slow down the growth of the disease. They also offer some survival benefits. But as the researchers state, these drugs do not cure. The disease will still progress, and people will die.
Amylyx continues its work on making the drug available commercially states Joshua Cohen, its co-CEO. He is hopeful that Amylyx’s efforts succeed so that private insurers and provincial programs will begin to sell the drug.