Treatment for Phenylketonuria Gains Approval in the EU

According to a story from CRWE World, the drug company BioMarin Pharmaceutical, Inc. recently announced that Palynziq, a treatment developed by the company for phenylketonuria, has earned marketing authorization from the European Commission. Palynziq has marketing authorization for a maximum dose of 60 mg for phenylketonuria patients who are unable to manage their phenylalanine concentrations using other available management techniques and therapies.

About Phenylketonuria (PKU)

Phenylketonuria (PKU) is a type of metabolic disorder affecting the enzyme phenylalanine, which builds up in the body and can cause serious problems. Normally, phenylalanine is broken down, but a mutation affecting the PAH prevents this from happening in patients with the disorder. Prompt treatment is essential to avoid major complications. Symptoms of phenylketonuria include small head size, low birth weight, an unusual musty odor, pale skin, behavioral problems, intellectual disability, heart issues, seizures, and mental disorders. All of these symptoms can be prevented with proper and timely treatment; phenylketonuria is routinely screened for in newborns for this reason. The primary management strategy is a highly controlled diet that is low in phenylalanine. Supplements may also be considered. Most patients eventually go off the diet when they reach adulthood. Some older patients may still have trouble managing the condition later in life. To learn more about phenylketonuria, click here.

About Palynziq

Palynziq effectively serves a substitute enzyme that helps the body break down and process phenylalanine. This is the second therapy that BioMarin has developed for phenylketonuria. Palynziq has previously gained approval in the US in May of last year for the same indication. The EU approval comes following the results of a Phase 3 clinical study that demonstrated that the drug was capable of sustaining reduced blood phenylalanine levels in phenylketonuria patients. The drug was also able to alleviate mood symptoms and inattention associated with elevated blood phenylalanine.

This approval will give patients in the EU another treatment option for their condition if they are unable to control their phenylalanine levels using other management approaches.


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