According to a story from PR Newswire, the pharmaceutical company BioMarin Pharmaceutical Inc. has recently announced that the U.S. Food and Drug Administration as issued approval for their product pegvaliase-pqpz, which will be marketed and branded as Palynziq. The injection is approved for the treatment of patients with phenylketonuria. Palynziq demonstrated its ability to bring down levels of blood phenylalanine (Phe) in clinical trials. Patients with the disease have uncontrollable levels of Phe, and Palynziq is indicated for patients with Phe levels over 600 micromol/L.
Phenylketonuria (PKU) is a metabolic disorder that is characterized by a poor ability or inability to metabolize Phe. The disorder is caused by a genetic mutation and presents symptoms soon after birth. However, babies with phenylketonuria appear healthy at birth assuming their mother does not have the disorder. Newborns are routinely screened for the disorder; it is essential to begin intervention before any damage can occur. Without treatment, patients experience mental disorders, seizures, intellectual disability, and behavioral issues. People with the condition often have musty body odor and pale skin. A mother with poorly treated PKU gives birth to children with low birth weight, heart issues, and a small head. Thankfully, with prompt treatment and management with dietary restrictions and medications, most patients will experience no effects and live normal lives. To learn more about phenylketonuria, click here.
Palynziq will be the second treatment developed by BioMarin for the management of PKU. The approval of the new drug is also timely, as May is National PKU Awareness Month. Patients now have the opportunity to celebrate a new and effective treatment option that will allow them to control their Phe levels more effectively. Some PKU patients still struggle to control their Phe levels even with appropriate dietary constraints and the use of currently available medications, so hopefully Palynziq will offer relief to this group.
BioMarin is slated to begin marketing and promotion of the new treatment immediately, and Palynziq is expected to be available for use by the end of June. The company also has plans to make the medication available outside of the U.S., and submitted a Marketing Authorization application to the European Medicines Agency this past March.
We believe rare disease patients are people, not a diagnosis. Through education, awareness and some humor, we help patients, caregivers and support persons by providing relevant and often inspirational news and stories.
Our goals are to share stories, cultivate strong community, provide the latest medical findings, connect people and pioneer production of patient worthy information. Help us attain these goals by telling us a little bit about yourself!
We’ve heard your feedback. Now, by creating a login below, you can customize your homepage to the rare conditions that are most important to you. You can also share your rare story directly through the site, so being part of our community is easier than ever!