BioMarin

This sponsor has funded 13 studies across 39 countries.

PAS funded by this sponsor.
EU PAS ID Status Title Protocol uploaded Results uploaded
6106 Finalised Lambert-Eaton Myasthenic Syndrome (LEMS) Registry (LEMS-01) No No
6835 Ongoing Morquio A Registry Study (MARS) Yes No
17347 Finalised 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 Kuvan® Adult Maternal Paediatric European Registry (KAMPER) No Yes
19286 Ongoing MPS VI Clinical Surveillance Program (CSP) No No
29031 Ongoing Cerliponase alfa Observational Study No No
34992 Ongoing A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (pegvaliase) in Subjects with... No No
35156 Ongoing A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (pegvaliase) During Pregnancy and Breastfeeding... No No
41426 Ongoing A Multicentre, Observational Study to Evaluate the Real-World Outcomes of Palynziq® (Pegvaliase) in Subjects with Phenylketonuria (OPAL) Yes No
47514 Ongoing 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 World Federation of Hemophilia Gene Therapy Registry (WFH GTR) No No
49071 Planned GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with ROCTAVIAN™ (valoctocogene roxaparvovec) (GENEr8-COAS... No No
49243 Ongoing 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.