June 17, 2014 4:55 am
Updated: June 17, 2014 4:56 am

Young girl may get much-needed cystic fibrosis drugs as provinces reach deal

Madi Vanstone (right) and her mother Beth (left) speak to reporters at Queen's Park on March 3, 2014.

Global News

TORONTO – Madi Vanstone, the young girl who lobbied Premier Kathleen Wynne for an expensive cystic fibrosis drug may soon have her treatment covered by OHIP.

Vertex Pharmaceuticals Incorporated says it has signed a letter of intent with the pan-Canadian Pricing Alliance that will enable public reimbursement of the drug Kalydeco.

The parties have been in protracted negotiations over the price of the drug, which reportedly costs more than $300,000 a year.

Vertex says about 100 Canadians with cystic fibrosis are candidates to use this drug.

WATCH: Previous stories about Madi Vanstone. 

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People who could be helped by the oral medication have one of a number of mutations on the CFTR gene.

The statement from Vertex says that each province and territory still must decide to reimburse the drug through its own drug plan.

The agreement does not cover Quebec, which does not take part in the pan-Canadian Pricing Alliance process.

Cystic fibrosis is caused by a defective or missing CFTR protein, the result of a mutation in the CFTR gene. Only children who inherit two faulty CFTR genes – one from each parent – have the disease. There are more than 1,900 known mutations in the CFTR gene.

People with CF have a poor flow of salt and water into and out of the cells of a number of organs, including the lungs. This results in a buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

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