Pegvaliase

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

  1. abdominal pain
  2. acneiform rash
  3. alopecia
  4. anaphylactic shock
  5. anaphylactoid reactions
  6. angioedema
  7. antibody formation
  8. anxiety
  9. arthralgia
  10. arthropathy
  11. atopic dermatitis
  12. back pain
  13. blepharedema
  14. bronchospasm
  15. conjunctivitis
  16. contact dermatitis
  17. cough
  18. diarrhea
  19. dizziness
  20. dyspnea
  21. ecchymosis
  22. edema
  23. erythema
  24. fatigue
  25. flushing
  26. headache
  27. hematoma
  28. hypotension
  29. hypoxia
  30. infection
  31. injection site reaction
  32. maculopapular rash
  33. musculoskeletal pain
  34. nasal congestion
  35. nausea
  36. panniculitis
  37. paresthesias
  38. petechiae
  39. pharyngitis
  40. pruritus
  41. psoriaform rash
  42. rash
  43. serious hypersensitivity reactions or anaphylaxis
  44. serum sickness
  45. sinusitis
  46. skin hyperpigmentation
  47. skin hypopigmentation
  48. syncope
  49. urticaria
  50. vomiting
  51. 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
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