Health minister intervenes to get drug approved for child with genetic disease

The health minister has approved a drug for a five-year-old child who has a rare genetic disease, and will ask the federal government for assistance to get a second medication for him.

The Steinbach family also learned Wednesday that a local couple, who wish to remain anonymous, will use their connection to Minnesota’s renowned Mayo Clinic, to help Leo Dupont get the drug he needs to slow down the progression of his illness.

Health Minister Uzoma Asagwara said Dupont’s family has been told Leo will soon have access to Miglustat, one of the drugs used to treat Niemann-Pick Disease, Type C.

“I have provided approval for the Health Canada-approved Miglustat,” Asagwara said on Wednesday.

“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.”

Asagwara said it’s not known yet whether the family will have to pay a portion of the cost, but it will be worked out with Manitoba Health’s non-insured benefits.

“Providing approval is the big piece and now there are a few more pieces which need to fall into place, but they will not be going through it alone,” the minister said.

As for the other drug the family and his doctor says the child needs, Miplyffa, Asagwara said that’s more difficult.

“The medication has not yet been approved by Health Canada and actually has also not been brought forward to Canada by the manufacturer,” the minister said.

“It means it can’t be sold or provided anywhere in Canada (and) without federal approval, we can’t fund or provide the drug… I’m writing to Health Canada asking them to engage with the manufacturer.”

Zevra Therapeutics, the manufacturer of Miplyffa, didn’t respond to a request for comment.

Asagwara, called the family’s situation “heartbreaking.”

“I can’t thank them enough for being courageous enough to share Leo’s story, to bring Manitobans into their family’s conversation, and for giving our government the opportunity to get to know them a little bit, too and to provide them with the approval we are able to provide.”

In June, Dupont was diagnosed with the genetic disease in which the body doesn’t have the protein it needs to process cholesterol properly. The buildup of cholesterol in the body’s cells leads to cognitive problems, as well as difficulty walking, swallowing and hearing, and ultimately results in death, usually by age 13.

It is diagnosed in one out of every 150,000 people annually.

The family went public this week because they hadn’t heard back from the province since Aug. 21, when their doctor submitted a request for Miglustat to be approved through the exceptional drug status program.

The family said while the disease is ultimately fatal, the drugs will allow him to have a better quality of life and slow down the progression of the illness.

Leo’s mom, Kayla Krentz Dupont, said she is thankful for the health minister’s help.

“We see this as a positive step. We still haven’t had an answer yet when he will give access to it. The minister was not able to provide a time frame,” she said.

When told about the philanthropist couple’s offer, Krentz Dupont began to cry.

“This is an answer to our prayers,” she said, her voice breaking, as the email was read to her. “This is the home run we have been swinging for. As Leo would say, this is the ‘Springer Dinger.’”

The child is a huge fan of the Toronto Blue Jays and uses the expression fans use to describe player George Springer’s clutch home run that got the team into the World Series.

In her reply email to the couple, Krentz Dupont said their offer to help “has been our dream, goal and prayer for our son after learning about his diagnosis in June of this year.”

The couple said they hope to introduce the family to a specialist at the Mayo Clinic.

“Additionally… we may be able to assist them in getting Miplyffa through some of our many contacts at the Rochester clinic,” the husband said.

“Thank you for sharing their story. It’s heartwrenching to see what they are going through.”

kevin.rollason@freepress.mb.ca