In accordance with paragraph 8 of Section IV of the Procedure for Expert Evaluation, the applicant must amend the instructions for medical use of the medicinal product upon receipt of new data, including bringing the instructions for medical use into line with the approved instructions for medical use of the original (reference) medicinal product, and ensure that the information on the medicinal product corresponds to current scientific knowledge.
The European Medicines Agency (EMA) has published the minutes of the meeting of the Pharmacovigilance Risk Assessment Committee (PRAC) held on June 08 – 11, 2026. Following the meeting, a decision was made that safety-related changes need to be introduced to the registration materials / instructions for medical use of medicinal products containing laronidase.
Laronidase – ALDURAZYME
Scope: Submission of the final report on the results of the post-authorization safety study PASS ALID01803, specified in the risk management plan (RMP) as a category 3 study. This is an observational open-label study of the effect of the use of the medicinal product ALDURAZYME (laronidase) on lactation in postpartum women diagnosed with mucopolysaccharidosis type I, and on their breastfed infants. The aim of the study is to establish whether laronidase activity is present in the breast milk of mothers with mucopolysaccharidosis type I and whether the medicinal product ALDURAZYME (laronidase) affected the growth, development and immune response of breastfed infants. Version 2.0 of the RMP was also submitted.
As stated in the RMP for the medicinal product ALDURAZYME (laronidase), the registration certificate holder (RCH) conducted a non-interventional PASS study, which was not mandatory under a requirement of the regulatory authority, in order to study the effect of the use of ALDURAZYME (laronidase) on lactation in postpartum women diagnosed with mucopolysaccharidosis type I, and on their breastfed infants. The Rapporteur assessed the final study report, as well as the RCH’s responses to the request for supplementary information.
Summary of recommendations and conclusions
- Based on the available data, the registration certificate holder's responses to the request for additional information, and the assessment of the PRAC rapporteur, PRAC concluded that the ongoing variation procedure regarding the evaluation of the final study report can be recommended for approval.
- The PRAC concluded that the instructions for medical use should be updated by changing the wording in the section "Use during pregnancy or breastfeeding" regarding breastfeeding / lactation, in order to reflect the insufficiency of data on the excretion of laronidase into breast milk, as well as the absence of reports of adverse reactions in a very limited number of breastfed infants whose mothers used the medicinal product ALDURAZYME (laronidase) during breastfeeding. In addition, when deciding on breastfeeding during treatment with the medicinal product ALDURAZYME (laronidase), the benefit of breastfeeding for the mother and the infant, the benefit of treatment for the mother, and the potential risk of adverse reactions in the breastfed infant should be taken into account.
Comprehensive and up-to-date information on an individual medicinal product (summary of product characteristics, post-registration procedural steps, RMP summary, additional risk minimization measures, etc.) that is registered under the centralized procedure can be found in the Human medicines European public assessment report (EPAR) on the EMA website.
Перелік лікарських засобів, що містять ларонідазу (laronidase) та дозволені до медичного застосування в Україні (за даними Державного реєстру лікарських засобів станом на 15.09.2026).
TO ANALYZE THE PUBLISHED INFORMATION AND SUBMIT THE CORRESPONDING APPLICATION FOR AMENDMENTS TO THE REGISTRATION MATERIALS, THE APPLICANT MUST USE THE ORIGINAL TEXT AVAILABLE AT THE FOLLOWING LINK:
