B.C. to cover $1-million-per-year drug for Victoria girl, 3, with rare, fatal disease

A Victoria family says the province has agreed to cover a life-saving, million-dollar drug for their youngdaughter.

The parents of Charleigh Pollock, 3, saidtheprovince has granted them access to Brineura, a promisingnew medication that has received federal approval but is notcovered byB.C.'s public health system.

Their daughter suffers from CLN2 or Batten disease, a rare and fatal genetic disorder that results in multiple seizures each day and eventually leads to brain damage.

She is the only person inBritish Columbia with the disease and one of 13 childrenin Canada with the condition, the family says. Most children who have it die between the ages of six and 12.

The case went before the province's independent drug review committee, which agreed to fund the treatment for Charleigh.

"We are in shock. We're excited. We're nervous. We have every emotion going on right now," said Jori Fales, Charleigh's mother, in an online video posted Friday.

The case highlights a growing classof drugs that the B.C. health ministry has described as "expensive drugs for rare diseases."

In Oct. 2018, the province covered the cost of 16 expensive drugs for rare disease, with list prices ranging from $100,000 to $3 million.

Theministry declined to comment on Charleigh's case, citing patient privacy.

'The first step'

Charleigh's family learned of her diagnosis three weeks ago and launcheda GoFundMe page to raise funds for the costly treatment, also known as enzyme replacement therapy.

Patients withCLN2are missing an enzyme which leads to brain damage. Brineura,administered every two weeks through a tube into the brain, helps replace thatmissing enzyme.

The treatment is new and costs more than $1 million a year. But clinical trials have shown promising results, the family says.

It will slow Charleigh's rapid deterioration until a cure is found, they say.

The girl suffered anunexplained seizure, the first sign of the disease,eight months ago.

As the diseaseworsens, a child will go blind and lose theability to walk and speak. They eventually develop dementia and lose all cognitive function.

So far, Charleigh, who's a few weeks shy of her fourth birthday, is still walking and can say a few words. She's being nourished with a feeding tube.

She will have to endure infusions and brain surgery in the months ahead, said Trevor Pollock, Charleigh's father, in the video.

"This is the first step in a long journey ahead for our family."