British Columbians under 18 living with a rare, genetic and chronically debilitating bone disorder will soon have free access to medication provided by the provincial government.
Symptoms of X-linked hypophosphatemia (XLH) develop around the age of one or two and include rickets, delayed walking, bowed legs, bone pain, impaired growth and severe dental issues. Adults with XLH can have symptoms such as bone and joint pain, fractures, severe dental issues, hearing loss and fatigue.
The province announced, in a media release, it will cover the cost of the drug burosumab, used to treat XLH, through its BC Expensive Drugs for Rare Diseases (EDRD) process. The drug’s annual cost is anywhere from $130,000 to $1.17 million, depending on the patient’s weight and age, according to the province.
Treatment usually starts at the time of diagnosis and continues until bones stop growing. It includes phosphate supplements combined with high-dose calcitriol. Growth hormones may also be required, as well as orthopedic surgery.
Clinical trials of burosumab in patients under the age of 18 showed reduced deformities in lower extremities, as well as significant improvements in stiffness and the severity of rickets.
There are approximately 15 people with XLH under the age of 18 in B.C., according to the province.
EDRDs are considered non-benefits, but some drugs and patients may be eligible for coverage on a case-by-case, last-resort basis. Patients will need to discuss their eligibility with their health-care provider.