Brazilian court orders government to supply unapproved drug

In a recent decision, the Federal Court of the First Region in the city of Brasilia ordered the Federal Government to provide NAGLAZYME to a Brazilian citizen, notwithstanding the fact that this drug did not have the necessary market approval to be sold to and/or used by Brazilian patients, regularly issued by ANVISA (Brazilian FDA).

NAGLAZYME is an enzyme replacement therapy used for the treatment of Maroteaux-Lamy syndrome (mucopolysaccharidosis VI, MPS VI), a rare, inherited and progressive metabolic disease that may lead to a severe disability, which affects approximately 1,100 persons in the world.  Among the arguments raised in court by the Federal Government for not providing NAGLAZYME, it stated that (i) NAGLAZYME did not have ANVISA’s market approval, (ii) there was no cure for Maroteaux-Lamy syndrome, (iii) this medicine was approved by the EMEA and FDA without the necessary clinical trials (iv) a six month treatment would almost cost US$182,700.

In spite of these arguments, the Court ruled otherwise and ordered the Federal Government to provide NAGLAZYME to this patient, on the grounds that the commercialization of the drug was already approved by the EMEA in Europe and by the FDA in the US.  It also pointed out that the risks resulting from the use of the NAGLAZYME not yet approved by ANVISA are, no doubt, less than the risks from not receiving any type of treatment, especially to those patients which suffer from terminating disease.

This decision represents a major breakthrough in the Brazilian legal system and may represent a leading case for other Brazilians with similar illnesses.