BioMarin

This sponsor has funded 13 studies across 39 countries.

PAS funded by this sponsor.
EU PAS ID Status RMP requirement Title Protocol uploaded Results uploaded
6106 Finalised EU RMP category 3 Lambert-Eaton Myasthenic Syndrome (LEMS) Registry (LEMS-01) No No
6835 Ongoing EU RMP category 1 Morquio A Registry Study (MARS) Yes No
17347 Finalised Not included in RMP The Impact of Achondroplasia on Quality of Life, Healthcare Resource Use, Clinical, Socio-economic and Psychosocial state of the Individual (LIAISE) No No
17360 Finalised EU RMP category 3 Kuvan® Adult Maternal Paediatric European Registry (KAMPER) No Yes
19286 Ongoing EU RMP category 3 MPS VI Clinical Surveillance Program (CSP) No No
29031 Ongoing EU RMP category 2 Cerliponase alfa Observational Study No No
34992 Ongoing EU RMP category 3 A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (pegvaliase) in Subjects with... No No
35156 Ongoing EU RMP category 3 A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (pegvaliase) During Pregnancy and Breastfeeding... No No
41426 Ongoing Not included in RMP A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria (OPAL) Yes No
47514 Ongoing EU RMP category 3 A multicentre, non-interventional study to evaluate long-term safety in patients with achondroplasia treated with Voxzogo® (vosoritide) (BMN 111-603) No No
48683 Planned Not included in RMP World Federation of Hemophilia Gene Therapy Registry (WFH GTR) No No
49071 Planned EU RMP category 1 GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec) (GENEr8-COAS... No No
49243 Ongoing EU RMP category 1 GENEr8-GTR: A Retrospective Cohort Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec): An Analysis of Patient Registries (BMN... No No

PAS by Risk Management Plan (RMP) requirement

BioMarin

The RMP for a medical product outlines strategies to identify, assess, and mitigate risks, including PAS for further evaluation.

Category 1 studies are imposed as condition of marketing authorisation.
Category 2 studies are imposed as specific obligation of marketing authorisation.
Category 3 studies are required under other circumstances.