Original Title: Pegvaliasa en fenilcetonuria

Authors' recommendations: No evidence on the use of pegvaliase in patients with phenylketonuria non-eligible for sapropterin has been found. Low-quality evidence on adding pegvaliase to diet in patients with phenylketonuria and poor metabolic control suggests that its use would reduce the levels of phenylalanine in blood and improve the cognitive-behavioral characteristics of patients at long-term. However, these clinical benefits should be considered also taking into account the occurrence of serious adverse events such as anaphylaxis, which have been reported in up to 2.7% of patients who received this treatment, and it had to be discontinued. The clinical practice guidelines consulted that mention the use of pegvaliase, indicate that its use should be assessed individually for each patient. As regards, coverage policies, the public funders from Germany cover it, as well as the private funders from the United States consulted. Other public funders from Europe consider that the evidence available is not enough as to issue a coverage recommendation. In Argentina, it is not commercially available because it has not been approved; therefore, its coverage is not included in the Mandatory Medical Program and its cost is not recovered through the Unique Reimbursement System either. The cost-effectiveness of this drug is uncertain in Argentina.

Project Status: Completed

Year Published: 2020

URL for published report: https://www.iecs.org.ar/home-ets/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Argentina

Organisation Name: Institute for Clinical Effectiveness and Health Policy

Contact Address: Dr. Emilio Ravignani 2024, Buenos Aires - Argentina, C1414 CABA

Contact Name: info@iecs.org.ar

Contact Email: info@iecs.org.ar

Copyright: Institute for Clinical Effectiveness and Health Policy (IECS)