Playnziq is a rare medicine containing pegvaliase-pqpz is a phenylalanine-metabolizing enzyme made up of recombinant phenylalanine ammonia-lyase (rAvPAL) conjugated to N-hydroxysuccinimide (NHS)-methoxy polyethylene glycol.
Phenylketonuria is caused by a defect in the PAH gene, a rare genetic condition that causes an amino acid known as phenylalanine to increase in the body.
Palynziq is used to decrease blood phenylalanine concentrations in adult patients suffering from phenylketonuria(PKU), in which patients have uncontrolled blood phenylalanine concentrations greater than 600 micromol/liter on existing management.
Palynziq medicine is a clear to a bit opalescent, colorless to a pale yellow solution available in the form of injection as a prefilled syringe in the strengths of:
One should consult such doctors who have experience in managing PKU disease, and administration of dosage is always according to the doctor’s prescription.
Doctors should obtain baseline blood phenylalanine concentration before starting the treatment.
The recommended initial induction dosage of the medicine is 2.5 mg via hypodermic injection once a week for 4 weeks.
One should titrate the medicine dosage step by step, according to the tolerability, over at least 5 weeks to achieve a 20 mg dose subcutaneously once a day.
To achieve a therapeutic response, titrate an effective maintenance dosage of the medicine to the patient. Professionals should use the lowest effective and tolerated dosage of the drug.
One should assess patient tolerability, blood phenylalanine concentrations, and dietary protein and phenylalanine intake during the treatment.
Discontinue the medicine pegvaliase in patients who have not achieved a response after 16 weeks of repeated treatment with the maximum dosage of 40 mg once a day.
Side effects of pegvaliase-pqpz injection for PKU are classified as:
Injection site reactions, arthralgia, hypersensitivity reactions, headache, skin reactions, pruritus, nausea, abdominal pain, oropharyngeal pain, and fatigue are common side effects of treatment with Palynziq.
Vomiting, coughing, and diarrhea are uncommon side effects of the medicine.
Anxiety, alopecia, and nasal congestion are less frequent side effects of taking this medicine.
Note: Every patient doesn’t need to experience these symptoms.
During induction/titration/maintenance of phenylketonuria treatment, 9% of patients experienced anaphylaxis. According to reports of clinical trials, symptoms of anaphylaxis are syncope, hypotension, hypoxia, dyspnea, wheezing, chest discomfort/chest tightness, tachycardia, angioedema, etc.
According to the patient’s tolerability, one should consider premedication with an H1-receptor antagonist, H2-receptor antagonist, or antipyretic before Palynziq administration.
Palynziq is available only via a restricted program under a REMS after phenylketonuria diagnosis.
Palynziq REMS program
Palynziq is available only via a restricted program under a REMS( Risk Evaluation and Mitigation Strategy) known as Palynziq REMS because of the risk of anaphylaxis in patients with phenylketonuria enzyme deficiency.
Requirements of the Palynziq REMS are:
The prescriber/professional must be certified with the program by enrolling in the program and completing training.
The prescriber must prescribe auto-injectable epinephrine with the medicine.
One should go for the certified pharmacies with the program, and the pharmacies must dispense only to patients authorized to receive it.
The patient must enroll in the program. One should educate the patient about the risk of anaphylaxis to ensure they understand the risks and benefits of treatment with the medicine.
The patient must have auto-injectable epinephrine available at all times while taking this medicine.
Other Hypersensitivity Reactions
Other than anaphylaxis, 69% of patients experienced different hypersensitive reactions during PKU treatment with pegvaliase. Healthcare professionals should consider premedication with an H1-receptor antagonist, H2-receptor antagonist, or antipyretic before the medicine administration based on the patient’s tolerability.
One should manage hypersensitivity reactions according to the severity of the response, recurrence of the reaction, and the patient’s clinical status, and may include dosage adjustment, temporary drug interruption, or treatment with antihistamines antipyretics, or corticosteroids.
Note the date removed from refrigeration on the carton.
Once stored at room temperature, do not restore the product in the refrigerator.
The shelf-life of the medicine expires after storage at room temperature for 30 days or after the expiration date on the product carton
Playnziq is a rare medicine containing pegvaliase-pqpz is a phenylalanine-metabolizing enzyme made up of recombinant phenylalanine ammonia-lyase (rAvPAL) conjugated to N-hydroxysuccinimide (NHS)-methoxy polyethylene glycol. Palynziq is used to decrease blood phenylalanine concentrations in adult patients. Phenylketonuria symptoms are stunted growth, tremors, trembling and shaking, hyperactivity, a musty odor of their breath, skin, urine, etc.
Palynziq contains pegvaliase-pqpz as an active ingredient, and sodium chloride, trans-cinnamic acid, tromethamine, and tromethamine hydrochloride are inactive ingredients.
For Palynziq, Biomarin is the innovator. Ikris Pharma Network is an authorized partner of it, who can help access this medicine in India for the patient via the legal procedure. To know Palynziq cost and more, contact Ikris at toll-free no. 18008891064 or write at firstname.lastname@example.org.
In India, you can buy Palynziq with the help of Ikris Pharma Network, an authorized partner of “Biomarin” for this medicine.