Last week I talked about Kuvan being a treatment for PKU. I'm a responder to Kuvan and I can speak to how well it works for me!
Pegvalise is another treatment for PKU, however I've never tried it, so everything below will just be information I've heard from other people or read online about it, as I have no personal experience with it. This is merely an information hub for those who are looking for information about it, or to inform people who don't have PKU about the different options that are available to us!
"Pegvaliase is a medication for the treatment of the genetic disease phenylketonuria. Chemically, it is a pegylated derivative of the enzyme phenylalanine ammonia-lyase that metabolizes phenylalanine to reduce its blood levels. It was approved by the Food and Drug Administration for use in the United States in 2018."
That's a lot of big words. Basically, it's an injection that helps lower blood levels in PKU patients by providing therapy to the enzyme. (See my blog post on blood levels titled "I am NOT afraid of needles"!) The company that distributes Pegvaliase is called Palynziq.
The drug is administered through many at home injections. There are a lot of different side effects that come from this drug just like every other medication, however I've heard of many people having to take allergy medicine with their Palynziq because of the anaphylactic shock that comes with each injection.
Although it is approved in the United States, it has not yet been approved in Canada, which may be partly why I haven't tried it yet. There is a lot of politics that comes with new drugs trying to be incorporated into active treatment of not just PKU patients, but any patient needing drugs for treatment. There are many tests that drug companies have to do and a lot of hoops to jump through before it is approved in a certain country.
The financial post states: "No clinical trials for Palynziq took place in Canada and Canada will not be in the next set of countries for Palynziq marketing applications, as Kuvan was and other new drugs for both common and rare disorders often are. The reason: delayed or no Kuvan reimbursement in Canada and the obligation to provide Kuvan to trial patients for many years to come."
As I read the testimonies of people who have tried it, it seems that a lot of people take a long time to start responding. If you read my Kuvan blog post, you'll remember that it only took me 3 days for my levels to drop significantly. It sounds like most people respond to this type of treatment, it's just a matter of how long it takes to do so. This is a benefit for people who don't respond to Kuvan, because only certain kinds of PKU patients respond to it.
As I said my knowledge of Palynziq is pretty limited so if you have experienced Palynziq drop some information in the comments to help each other out!
In the meantime, check out this link for more information:
