Loading articles...

Parents fight to get daughter costly life-changing drug

Last Updated Jan 23, 2018 at 8:33 pm EDT

The family of a three-year-old Peterborough girl is fighting to get coverage for an expensive new drug they say will help her cope with a disease that is taking away her ability to move.

Khloe Mossor has been diagnosed with Type 2 Spinal Muscular Atrophy. Known as SMA, it’s a rare degenerative disorder that destroys all of the body’s muscles.

“I’d like to walk someday,” Khloe said.

Today, she can’t sit up on her own and must use a wheelchair.

There is a treatment available, called Spinraza, but it costs US$750,000 in the first year, and US$375,000 a year for the rest of a patient’s life. It’s one of the most expensive drugs ever considered for public funding in Ontario.

For many, it is a miracle. But hope is fading for Khloe and other children with the same genetic disorder.

“It’s frustrating because it’s just a matter of when will Khloe’s time come?” said her dad Matt Mossor.

Spinraza is in the approval process for government coverage.  The first step is a review by the Canadian Drug Expert Committee, which does a cost-benefit analysis.

In December, the committee released recommendations about which SMA patients should be covered. Its conclusions are devastating to Khloe’s parents; only children who are under seven months of age and have Type 1 SMA, the most severe type, should receive provincial coverage for the drug. They must also be treated within 26 weeks after diagnosis. It means most SMA patients alive today would not be eligible because they are too old.

“Why only Type 1?” said Khloe’s mother Jessica Mossor. “A lot of kids aren’t diagnosed until after seven months old. They are narrowing it down to just a few.”

Biogen, the manufacturer of Spinraza, says the drug is available without such tight restrictions in 15 other countries, including the U.S., where it is available to all based on plan coverage.

Canada’s drug committee concludes there is “insufficient evidence” that Spinraza is effective or safe in other patients. That’s hard for Khloe’s parents to believe considering the miracles they see in other children, like three-year-old Toronto girl Sophia Gall, who has Type 3.

Sophia was able to ride a bike one week after her first injection of Spinraza and is steadily improving.

Khloe’s parents are still fighting for her but worry they are reaching a dead end.

“Kids are going to die in the process; they’re going to lose abilities in the process,” Mossor said. “It’s not fair. It breaks my heart.”

Related stories:

Family fights for access to life-changing, but prohibitively expensive drug

Family elated after girl with rare disorder gets funding for costly drug