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Compassionate Use

Access to Investigational Medicines

Arrowhead Pharmaceuticals, Inc. is committed to bringing new investigational medicines to patients with serious diseases as quickly and efficiently as possible. Arrowhead’s expanded access to investigational medicines is a means by which investigational medicines are made available, under certain circumstances, to treat patients with serious diseases who are ineligible or unable to participate in an ongoing Arrowhead clinical trial.  

As with any investigational medicine that has not been approved by regulatory authorities, the investigational medicine may or may not be effective in treating your diagnosis or condition, and there may be risks associated with its use. If you are a patient interested in participating, you should talk to your physician about the potential benefits and risks of taking part in the expanded access to an investigational medicine. 

Eligibility Criteria

Arrowhead requires the following criteria be met for a request to be considered: 

  • The investigational medicine must be part of an active clinical development program, 
  • Access to the medicine will not compromise clinical trials or the regulatory pathway, 
  • There is substantial scientific evidence to support the benefit risk profile of the investigational product for its intended use,  
  • Sufficient supply of the investigational medicine is available, and 
  • It is logistically possible to safely administer the investigational medicine outside of a clinical trial setting  

The patient and the treating physician must also meet the following criteria to be considered by Arrowhead for access to an investigational medicine: 

  • They have a serious or immediately life-threatening disease 
  • They lack other currently available therapeutic options  
  • They are unable to join an active clinical trial of the investigational product 
  • Their treating physician believes there is potential for the patient under consideration to reasonably expect benefit from the treatment  

If the above criteria are met, Arrowhead will consider an investigational medicine request from the patient’s treating physician subject to local/national laws and regulations. There is no guarantee to access of an investigational medicine.  

If the investigational medicine does not demonstrate a positive risk benefit to patients in ongoing clinical trials, the medicine will cease being made available.  

If you are a patient who is interested in requesting access to an Arrowhead investigational medicine, please speak with your physician.  

If you are a qualified and licensed physician who is seeking to request a patient receive access to an Arrowhead investigational medicine, please direct any questions or submit a request to Arrowhead Pharmaceuticals, Inc. at (626) 304- 3400 or email us at  

Additional information can be found by going to  

Receipt of a request will typically be acknowledged by Arrowhead within 5 business days. All requests will be evaluated in a fair and unbiased manner.

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