San Rafael-based BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) got the green light this week from the European Commission to begin marketing Palynziq, a drug it has been developing to treat a rare genetic disorder that can result in intellectual disability and psychiatric problems.
Last may the U.S. Food and Drug Administration (FDA) approved Palynziq Injections in certain adult patients with the disorder.
The drug treats a condition where an enzyme required for metabolizing an essential amino acid found in most protein-containing foods is not sufficiently produced by the body, causing those amino acids to accumulate to unsafe levels in the blood that become toxic to the brain, according to a statement from the company.
The approval took into account Phase 3 studies showing the drug resulted in lowered levels of the dangerous amino acids in a test group. BioMarin estimates there are 50,000 patients with the disorder in the regions where it operates.
The company’s first-quarter results last month showed a jump in net product revenues for several of its treatments amid a bigger net loss than a year before.
In a statement released with the results, the company attributed the increased loss to increased costs associated with the ongoing U.S. commercial launch and European Union prelaunch of Palynziq.
The drug maker showed a net loss of $56.5 million for the quarter ending in March, up from the $44.1 million loss in the first quarter of 2018.