Since ages, we are all aware of the fact that phenylketonuria has only one appropriate treatment. This treatment was a life-long adherence to a diet with a strict low value of phenylalanine. Well if you are still on such a strict diet, then we have got the good news for you.
Recently, the USFDA extended its approval to a drug named Palynziq to be employed in the treatment of phenylketonuria. The generic name for Palynziq is pegvaliase-pqpz. USFDA is a food and drug regulating authority in the United States.
What is PKU?
PKU stands for Phenylketonuria. It represents one of the rare genetic disorders. This disorder is characterised by the abnormal building up of the phenylalanine content in the human body. The phenylalanine belongs to the family of amino acids. This building up of the phenylalanine content in the body occurs when the body lacks its efficiency to create the phenylalanine hydroxylase. The phenylalanine hydroxylase is an enzyme. It is used to cleave the phenylalanine, and the cleaved phenylalanine then gets converted to the tyrosine.
The ending term ‘uria’ in the name Phenylketonuria is indicative of the remarkably higher levels of phenylalanine present within the human body. They are so high enough that they are easily traceable within the urine content of the concerned person.
Being a rare genetic disorder, PKU has a comparatively lower rate of prevalence. It affects about one in either ten thousand or fifteen thousand people in the United States. If you leave PKU untreated, it has got the calibre of severe psychiatric disabilities.
Treatment of Phenylketonuria with the Palynziq
Like every drug undergoes a trial before its launch in the actual market, the Palynziq’s safety and the efficacy too was subjected for examination under the clinical trials. The study involved people with higher levels of phenylalanine in their bloodstream.
To list a few of the side-effects were the reactions that occurred at the injection-sites, hypersensitivity reactions and other physical problems. The drug’s label claim of a lower risk of the anaphylaxis.
The BioMarin Pharmaceutical is the producer of Palynziq. It is a Novato, California based firm.