(624) Elexacaftor, tezacaftor, and ivacaftor for the treatment of cystic fibrosis: experience in Argentina from October 2021 to March 2022.

Background:

Introduction:



Elexacaftor, tezacaftor, and ivacaftor therapy for Cystic Fibrosis was registered in Argentina in October 2021 by the ANMAT (Administración Nacional de Medicamentos, Alimentos y Tecnología Médica - National Drug, Food and Medical Technology Administration), with a Risk Management Plan (RMP) and a 5-year Patient Monitoring Program (PMP).



Objectives:

To ensure patient care in a safe manner, strengthening knowledge about Pharmacovigilance and promoting the notification of adverse events among health professionals.

Methods: Observational study. The patients were included in the PMP after they signed an informed consent form. During the monitoring program, the treating physicians used a model form, previously approved by the ANMAT, for the follow-up of the patients per visit and the notification of adverse events.

Results: Since the product registration until March 2022, 167 patients were included in the PMP: 87 patients were female, and 80 were male, with an age range of 6 to 62 years, and an average age of 24.04 years.

Of the total number of patients who signed the informed consent form, as of March 2022, follow-up information was obtained from 127 patients treated with elexacaftor, tezacaftor, and ivacaftor: 51 patients were homozygous for F508 del, 73 were heterozygous for F508 del, and 3 patients had a different genotype.

During the follow-up period, 20 adverse events were registered, 2 of which were serious, related to treatment, and required treatment discontinuation: one of the events included hepatic manifestations and the other event included skin manifestations.

Conclusions: The elexacaftor, tezacaftor, and ivacaftor PMP provided information about native patients in real life, allowing a deeper understanding of highly effective modulator treatment in Argentina.

Although the follow-up period was short, it is expected that this PMP will allow us to obtain useful information for clinical practice.

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