- Access to Kuvan still needed as third component of PKU Comprehensive
Brain Protection Strategy -
VANCOUVER, Nov. 28, 2013 /CNW/ - Canadian PKU and Allied Disorders
(CanPKU) is delighted with the B.C. government's decision to provide
funding for special low-protein medical foods for patients affected by
Phenylketonuria (PKU) and similar inborn errors of metabolism (IEMs).
Medical foods play a critical role in preventing devastating
neurocognitive, psychiatric and physical symptoms, and in some cases
even death, caused by PKU and 24 other rare, inborn metabolic
disorders. The foods are one of three medical components of CanPKU's Comprehensive Brain Protection Strategy for People with PKU submitted to the B.C. government in February 2013.
In PKU, the body is unable to process an essential amino acid found in
dietary protein, called phenylalanine (or "Phe"), and the resulting
accumulation of Phe in the blood is toxic to the brain. If left
untreated, symptoms can range from mild cognitive impairment to severe
mental retardation. As such, patients must adhere every day of their
lives to a complicated and highly-restrictive low-protein diet to stave
off Phe's harmful impacts. Medical foods help PKU patients maintain
their blood Phe levels at acceptable levels, protecting their brains.
Patients with other IEMs are affected by different amino acids in
protein, and low-protein medical foods help them to maintain proper
growth while on the severely restricted diet. One such IEM called Maple
Syrup Urine Disorder (MSUD) can cause death if a similar diet is not
adhered to. Medical foods, which cost on average 10 times more than
regular foods, are not regulated by the Canadian Food Inspection
Agency, but instead by Health Canada as medical treatment.
"We commend B.C., and particularly the leadership of Health Minister
Terry Lake, for recognizing low-protein medical foods as a key
treatment to improve IEM health outcomes. This is a decision that will
go far to help the children and adults in this province who work hard
each day to protect their brains and lives from these diseases," says
Nicole Pallone, from Sparwood, B.C., who is vice president of CanPKU
and mother to a five-year-old daughter with PKU. "This announcement
marks B.C.'s most significant improvement in funding for PKU treatments
in 50 years - and our community is so thankful to all who contributed
to this decision."
According to the Ministry of Health, each patient in B.C. who requires
this treatment will be entitled to a subsidy of $250 per month to spend
on special medical foods, starting January 1, 2014. Prior to this
decision, B.C. only provided a nominal $40 monthly stipend for foods,
which was only available to patients on social assistance. Aside from
medical foods, B.C. IEM patients receive synthetic amino acid formulas
which continue to form the basis of the complicated medical diet. These
formulas are high in protein, vitamins and minerals, but are
specifically manufactured to exclude the amino acids that cause harm to
patients with each condition.
Access to third treatment in PKU Comprehensive Brain Protection Strategy still needed
While this funding decision marks a monumental step forward for PKU
treatment in the province, one critical treatment remains inaccessible
to patients who depend on B.C. Pharmacare. Kuvan (sapropterin
dihydrochloride), the first and only Health Canada approved drug
therapy for PKU1, is now funded in Ontario and Saskatchewan, as well as Quebec where the
government funds Kuvan on a case-by-case basis for women with PKU who
are pregnant or plan to become pregnant.2 Kuvan is covered for some patients with private health insurance.
Following the approval of Kuvan in 2010, negotiations with the drug's
manufacturer began with the drug programs in Ontario, Saskatchewan and
B.C. Much to the disappointment of PKU patients in B.C., it was the
only province to walk away from these negotiations. It remains unclear
to the patient community why B.C. funds Kuvan to treat BH4 deficiency
(originally called malignant PKU) and as a diagnostic tool to determine
whether newborns with high Phe levels have PKU or BH4 deficiency, but
not for its Health Canada approved indication to treat PKU.
"It is our sincere hope that B.C. continues to improve PKU patients'
health outcomes by providing access to the remaining essential
treatment tool - Kuvan - to ensure that the brains of adults,
adolescents and children are protected," says John Adams, President and
CEO of CanPKU, whose adult son has PKU and has been successfully
treated with Kuvan for more than six years. "We are grateful to the
Ministry of Health for its renewed support for patients and families in
B.C., but our mutual work is not yet done to bring treatment of PKU to
national and international standards."
To date, Kuvan is publicly funded and accessible to patients with PKU in
Austria, Belgium, Denmark, France, Germany, Greece, Italy, Japan,
Netherlands, Norway, Slovakia, Spain, Switzerland and the United
PKU (phenylketonuria) is a rare inherited, brain-threatening metabolic
disorder, observed when the body is unable to process phenylalanine
("Phe"), an essential amino acid found in dietary protein. The
resulting accumulation of Phe in the blood is toxic to the brain, and
if left untreated, symptoms can range from mild cognitive impairment to
severe mental retardation. Approximately 1 in 12,000 to 15,000 infants
in Canada is born with PKU. All provinces and territories, including
B.C., offer newborn screening tests to determine if a child is born
with PKU. If PKU is detected, the appropriate treatment must be
initiated immediately and maintained throughout life to ensure normal
About Canadian PKU and Allied Disorders Inc.
Canadian PKU and Allied Disorders Inc. is a non‐profit association of
volunteers, dedicated to providing accurate news, information and
support to families and professionals dealing with PKU and similar,
rare, inherited metabolic disorders. Our mission is to improve the
lives of people with PKU and allied disorders and the lives of their
families. By allied disorders we mean other rare, inherited metabolic
disorders also detected by newborn screening. For more information,
visit www.canpku.org and download our comprehensive resource for patients and families, PKU and the Brain.
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SOURCE: Canadian PKU and Allied Disorders Inc.
For further information:
President & CEO