Innovative therapeutics


Compassionate Use Policy

At BioMarin, we are committed to developing safe and effective therapies for rare diseases and providing those therapies to the broadest group of patients as quickly as possible. As part of that commitment, we support expanded access and compassionate use programs when we have substantial scientific evidence to support both the safety and the efficacy of an investigational product for an indication, and when it is logistically practicable.

BioMarin has a two-step process for determining whether the company will provide an experimental therapy under compassionate use.

In the first step, the company will evaluate whether:

  • there is substantial scientific evidence to support both the safety and the efficacy of a product for an indication, typically after positive Phase III data is available;
  • it has been established that access on a compassionate use basis will not compromise clinical trials or the regulatory pathway;
  • there is adequate supply of the experimental therapy; and
  • the compound can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.

If the company decides that, under the first step, compassionate use is appropriate and should be available as a general matter, then the company evaluates an individual’s request for compassionate use.

This second step uses the following criteria:

  • the relevant patient’s underlying medical conditions do not pose safety risks that have not been sufficiently studied;
  • the request has been made by a qualified and licensed physician with expertise and facilities appropriate for the administration of the therapy, monitoring, managing and reporting side effects, as well as patient experience;
  • the patient’s condition is of a type and stage commensurate with the indication for which the investigational therapy is likely to be approved;
  • the patient does not qualify for participation in ongoing or pending clinical trials; and all necessary regulatory/institutional approvals have been obtained to allow the administration.

Requests for access to experimental therapies must be made by a qualified and licensed physician through BioMarin Medical Information and will be referred to a core team for evaluation.

Contact BioMarin Medical Information



Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI) PKU, LEMS



Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

Global Medical Information

Contact Global Medical Information