Pegvaliase Brand Name– PALYNZIQ
What is Pegvaliase
Pegvaliase is a novel subcutaneous treatment used for the treatment of phenylketonuria (PKU) in adults who have uncontrolled blood phenylalanine (Phe) concentrations greater than 600 micromol/L despite current management strategies, such as medical foods and phenylalanine-restricted diets.
In the PRISM phase 3 studies, mean (SD) blood Phe was 1,232.7 (386.4) micromol/L at baseline, 564.5 (531.2) micromol/L at 12 months, and 311.4 (427) micromol/L at 24 months, a decrease from baseline of 51.1% and 68.7%, respectively. Within 24 months, 68.4% of participants achieved blood Phe 600 micromol/L or less, 60.7% of participants achieved blood Phe 360 micromol/L or less, which is below the upper limit recommended in the American College of Medical Genetics and Genomics PKU management guidelines, and 51.2% achieved blood Phe 120 micromol/L, below the upper limit of normal in the unaffected population.
Improvements in neuropsychiatric outcomes were associated with reductions in blood Phe and were sustained with long-term pegvaliase treatment.
Adverse events were mild to moderate and more frequent in the first 6 months of therapy and 96% resolved without dose interruption or reduction.
The most common side effects were arthralgia (70.5%), injection-site reaction (62.1%), injection-site erythema (47.9%), and headache (47.1%). Acute systemic hypersensitivity events were observed in 12 patients (17 events); of these, 6 participants remained on treatment.
Acute systemic hypersensitivity events including potential events of anaphylaxis were not associated with IgE responses, and all events resolved without sequelae.
Anaphylaxis occurred most frequently during upward titration of the dose within the first year of treatment.
Because anaphylaxis may occur, there is a boxed warning regarding this risk and the product is only available via a Risk Evaluation and Mitigation Strategy (REMS) called the Palynziq REMS Program.
Pegvaliase-pqpz was approved by the FDA in May 2018.
Indications
- phenylketonuria (PKU)
For the treatment of phenylketonuria (PKU) in patients with uncontrolled blood phenylalanine (Phe) concentrations greater than 600 micromol/L on existing management
Side Effects
- abdominal pain
- acneiform rash
- alopecia
- anaphylactic shock
- anaphylactoid reactions
- angioedema
- antibody formation
- anxiety
- arthralgia
- arthropathy
- atopic dermatitis
- back pain
- blepharedema
- bronchospasm
- conjunctivitis
- contact dermatitis
- cough
- diarrhea
- dizziness
- dyspnea
- ecchymosis
- edema
- erythema
- fatigue
- flushing
- headache
- hematoma
- hypotension
- hypoxia
- infection
- injection site reaction
- maculopapular rash
- musculoskeletal pain
- nasal congestion
- nausea
- panniculitis
- paresthesias
- petechiae
- pharyngitis
- pruritus
- psoriaform rash
- rash
- serious hypersensitivity reactions or anaphylaxis
- serum sickness
- sinusitis
- skin hyperpigmentation
- skin hypopigmentation
- syncope
- urticaria
- vomiting
- wheezing
Monitoring Parameters
- neurologic function
- serum phenylalanine (Phe) concentrations
Contraindications
- breast-feeding
- pregnancy
- requires an experienced clinician
- serious hypersensitivity reactions or anaphylaxis
Interactions
- Conjugated Estrogens; Medroxyprogesterone
- Estradiol Cypionate; Medroxyprogesterone
- Medroxyprogesterone