New Treatment Approved for Rare Disease PKU

Palynziq (pegvaliase-pqpz) has been approved by the U.S. Food and Drug Administration to treat phenylketonuria, commonly called PKU.

People with the rare yet serious inherited disorder can't break down an amino acid called phenylalanine, which is found in many sweeteners and protein-containing foods.

The disorder affects about 1 in 10,000 to 15,000 people in the United States. Left untreated, it can lead to serious learning, developmental and psychiatric disabilities, the FDA said in a news release.

Palynziq's safety and effectiveness were evaluated in clinical studies involving people with high concentrations of blood phenylalanine. The most common side effects of the enzyme substitution therapy were injection-site reactions, allergic-like hypersensitivity reactions, joint pain, headache, itchy skin, nausea, dizziness and abdominal pain.

The drug's label warns of a less-common risk of life-threatening anaphylaxis, which the agency said most often occurred when the dosage was increased during the first year of treatment.

Palynziq is produced by BioMarin Pharmaceutical, based in Novato, Calif.

More information

The FDA has more about this approval.


Copyright © 2013-2018 HealthDay. All rights reserved.

Mental Health Does Not Come With A Manual, It Comes With Friends & Family Support That Never Gives Up!
Join now

Related Content

What If Your Anxiety & Depression Is Really A Sign Of ADHD?
What If Your Anxiety & Depression Is Really A Sign Of ADHD?

More often than not, when a person over the age of 10 is diagnosed with attentio...

Read more
New Patent By OxyContin Maker May Treat Pain But Comes With Addictive Potential
New Patent By OxyContin Maker May Treat Pain But Comes With Addictive Potential

Purdue Pharma, the maker of OxyContin, has received a patent designed to treat o...

Read more
Why Are Transgenders Experiencing More Mental Health Issues Than The General Public?
Why Are Transgenders Experiencing More Mental Health Issues Than The General Public?

Individuals who identify as transgender tend to experience higher rates of menta...

Read more
New Research In Schizophrenia Shows That A Video Game Can Aid In Preventing Hallucinations
New Research In Schizophrenia Shows That A Video Game Can Aid In Preventing Hallucinations

People with schizophrenia can be trained by playing a video game to control the ...

Read more
I was 12 When I Survived 9-11 But My Peers & I Are Only Now Realizing Our PTSD
I was 12 When I Survived 9-11 But My Peers & I Are Only Now Realizing Our PTSD

As the Twin Towers burned and eventually collapsed on 9/11, I was running from m...

Read more
How Running Helped These Everyday People Beat OCD, Depression & Anxiety
How Running Helped These Everyday People Beat OCD, Depression & Anxiety

When documentary photographer Martin Eberlen was diagnosed with ADHD in his earl...

Read more