[10] In 2010, BioMarin was granted marketing approval by the European Commission for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate for the treatment of the rare autoimmune disease Lambert–Eaton myasthenic syndrome (LEMS).

[14] In 2012, BioMarin acquired Zacharon Pharmaceuticals, a private biotechnology company based in San Diego focused on developing small molecules targeting pathways of glycan metabolism.

[15] In 2014, BioMarin acquired a histone deacetylase inhibitor chemical library from Repligen for $2 million with the intention of advancing work toward therapies for Friedreich's ataxia and other neurological disorders.

[16] In November 2014, the company agreed to the acquisition of Prosensa for up to $840 million;[17] however, the range of treatments for Duchenne muscular dystrophy failed to attain FDA approval, and development ceased in May 2016.

The company states that prior to its licensing, there was no guaranteed quality control of the product and no way of formally monitoring for uncommon side effects through the regulatory process.

[24] On the advice of her doctor, Andrea Sloan, a patient with advanced ovarian cancer, requested that the company provide her with access to BMN 673, an unapproved PARP inhibitor drug candidate that had exhibited promising activity in a small Phase 1 clinical trial.

BioMarin stopped providing free Vimizim at the beginning of the year after negotiations with Belgian health authorities regarding reimbursement of the product repeatedly failed.