Health Canada Approves Ruzurgi for LEMS Patients Ages 6 or Older

Health Canada has approved Ruzurgi (amifampridine) for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in individuals ages 6 or older.

In Canada, the oral medication will be commercialized by Médunik Canada, per an agreement with Jacobus Pharmaceuticals, the original developer of Ruzurgi. 

“We are proud to have reached an exclusive distribution agreement to ensure Canadian patients can benefit from this life-changing medication,” Éric Gervais, executive vice-president of Médunik Canada, said in a press release. “Thus fulfilling, furthermore, Médunik Canada’s mission with the addition of a third product to our orphan disease treatment product offering.”

LEMS is caused by an immune system attack against a protein called voltage-gated calcium channel (VGCC), which is found at the sites where nerve cells communicate with muscles. This autoimmune attack decreases the ability of nerve cells to send chemical signals to muscles, resulting in muscle weakness.

Ruzurgi works by blocking proteins called potassium channels on neurons to give these cells more time for the remaining VGCCs to stay open and send signals. The improved communication from nerve cell endings may thereby increase muscle strength.

The U.S. Food and Drug Administration in 2019 approved Ruzurgi to treat children with LEMS ages 6 to 17. That decision was supported by clinical data collected from adults with LEMS, and simulations to identify the best dosing regimen for children. In particular, the U.S. regulatory agency analyzed data from a Phase 2 trial called DAPPER (NCT01511978), which showed that Ruzurgi lessened muscle weakness in people with LEMS according to objective assessments — most notably the Triple Timed-Up-and-Go (3TUG) test, which measures the time it takes a patient to get up from a chair, walk a short distance, and then return to the chair. The trial’s participants also reported less muscle weakness.

The notice of compliance issued for Ruzurgi by Health Canada, the country’s regulatory agency, comes shortly after the approval of another LEMS medication, Catalyst Pharmaceuticals‘ Firdapse (amifampridine).

Prior to Health Canada’s decision, Ruzurgi had been made available for people with LEMS through a Special Access Program. That program provides patients access to therapies that have not yet been approved for marketing, but are deemed to have the potential to treat serious conditions with unmet medical needs.

“Considering that many patients are already under treatment due to the Special Access Program (SAP), Médunik Canada has plans in place to ensure a smooth transition towards a dedicated Patient Support Program (PSP) to avoid any treatment interruptions,” said Dany Hallé, vice-president of commercial affairs at Médunik Canada.