Rare Disease Breakthrough: manitoba Boy Gains Access to Life-Slowing Medication After Public Appeal
WINNIPEG, MB – A Manitoba family’s desperate fight for access to a life-slowing medication for thier son’s rare genetic disorder has culminated in a victory, as the province has approved coverage for Miglustat. Théo Dupont, a child diagnosed with Niemann-Pick type C disease, will now be able to receive the treatment after his parents launched a public campaign to pressure the provincial government.
Niemann-Pick type C is a rare,progressive genetic disorder that affects multiple organ systems. For Théo, and others with the condition, Miglustat offers a potential to slow the disease’s progression.Though, the drug carries a meaningful price tag – approximately $100,000 per patient, per year. prior to Wednesday, Miglustat was not approved for use in Manitoba, leaving the Dupont family facing an insurmountable financial barrier.
“In two days, we had 2,500 emails sent to the Ministry of Health,” said Mario Dupont, Théo’s father, highlighting the swift response from the community.
The public pressure worked. Manitoba’s Minister of Health, Uzoma Asagwara, confirmed the approval. “We are happy that the family contacted us, we expedited the process and I accepted the authorization. We work with the family so they can get the medication as soon as possible,” Asagwara stated.
While the approval of Miglustat is a significant step, Dr. Philippe Campeau notes the high cost remains a challenge. Dr. Campeau is also exploring othre potential treatments, including Miplyffa, another drug recently approved in the United States. However, Miplyffa’s even higher price – nearly US$1.3 million, according to a 2024 Reuters article – makes Canadian approval unlikely.
“Once Health Canada has declared that these drugs are safe and effective, provincial committees (…) will evaluate their cost-benefit ratio to determine whether they should be reimbursed by public insurers, which could prove challenging given their high price,” Dr. Campeau explained.
Ultimately, Dr. Campeau believes the future lies in gene therapy, a potential cure that could halt the disease’s progression entirely. “We hope that our patients have access to as many medications as possible to the extent that pharmaceutical companies will offer medications at prices that are acceptable to public insurers,” he added.
