Health Minister Secures Drug Approval for Child with Rare Disease

The health minister of Manitoba, Uzoma Asagwara, has approved the use of a medication for five-year-old Leo Dupont, who suffers from a rare genetic disorder known as Niemann-Pick Disease, Type C. This decision marks a significant step in providing much-needed treatment for the child, whose condition affects the body’s ability to process cholesterol, leading to severe cognitive and physical challenges.

Asagwara announced on September 27, 2023, that Leo will soon have access to Miglustat, one of the medications approved by Health Canada for this condition. The minister emphasized that the government is committed to supporting the Dupont family through this process. “I have provided approval for the Health Canada-approved Miglustat,” said Asagwara. “The work is underway right now to ensure the department is working with the family to support them in the next steps so they will be able to start this medication.”

While the family awaits further details about potential costs associated with Miglustat, Asagwara assured them that Manitoba Health’s non-insured benefits will assist in clarifying any financial obligations. “Providing approval is the big piece,” Asagwara noted, indicating that they are working to ensure no steps are overlooked.

The situation is more complex concerning another medication, Miplyffa, which the family and Leo’s doctor believe is essential for his treatment. Asagwara explained that Miplyffa has not yet received approval from Health Canada and has not been made available in Canada by its manufacturer, Zevra Therapeutics. “Without federal approval, we can’t fund or provide the drug,” he stated, adding that he is formally requesting Health Canada to engage with the manufacturer to expedite the approval process.

Leo was diagnosed with Niemann-Pick Disease, Type C in June 2023. This genetic condition is diagnosed in approximately one in every 150,000 individuals and typically leads to severe health complications, often resulting in death by the age of 13. The disease is characterized by a deficiency in a critical protein that disrupts cholesterol metabolism, leading to harmful cholesterol accumulation in cells.

The Dupont family has been vocal about their struggle to secure timely treatment. They went public with their challenges after not receiving updates since August 21, when their doctor submitted a request for Miglustat through the exceptional drug status program. Leo’s mother, Kayla Krentz Dupont, expressed gratitude for the health minister’s intervention, stating, “We see this as a positive step,” although she noted that they still do not have a timeline for when Leo will begin treatment.

In an uplifting turn, the family recently learned that a local couple, who wish to remain anonymous, plans to leverage their connections with the renowned Mayo Clinic in Minnesota to assist Leo. This couple hopes to introduce the family to a specialist and potentially facilitate access to Miplyffa through their contacts at the clinic. Kayla expressed her emotional response to the couple’s offer, saying, “This is an answer to our prayers.”

As the Dupont family navigates these medical challenges, the support they have received from both the government and the community highlights the importance of collaboration in addressing rare genetic disorders. Asagwara described the family’s situation as “heartbreaking” and commended them for their courage in sharing Leo’s story. This collective effort aims not only to improve Leo’s quality of life but also to raise awareness about the urgent need for accessible treatments for rare diseases.