Access to Our Therapies

Above all, our goal is to ensure that patients have the opportunity to achieve the best possible therapeutic outcomes. We invest in broadening access to our therapies to meet the needs of patients, in addition to offering personalized and comprehensive support services – at no cost.

We work closely with the patient community to ensure that all who are medically eligible for our therapies have affordable access to Thiola®(tiopronin) tablets, Cholbam® (cholic acid) capsules, and Chenodal® (Chenodiol Tablets 250 mg).

Our Total Care HUB® is a free support program for people who have been prescribed Retrophin therapies. This program eases access to therapy, while providing live, on-demand support from dedicated pharmacists and HUB counselors to answer product questions, and assist with insurance verification, reimbursement options, and financial support.

We offer co-pay assistance and provide free medication to those who qualify, as part of our patient assistance program. We also sponsor free cholestatic genetic testing for patients who qualify to help diagnose a variety of disorders.

Below, you can learn more about our Total Care HUB® services exclusively for patients prescribed a Retrophin therapy:


Compassionate Use/Expanded Access

We are focused on making our investigational therapies available to as many eligible patients as we can – and as quickly as possible. To ensure the broadest possible access to these therapies, we conduct clinical trials supporting regulatory approvals from the FDA and other health authorities around the world.

While we believe that participating in a clinical trial is the best way for patients to access our investigational therapies, we recognize and understand that there are certain patients battling life-threatening disorders who may not be eligible for our clinical trials, or have a critical need for immediate care outside of a clinical trial.

For patients in these extreme situations, we accept requests for special access to our investigational therapies, which is often referred to as compassionate use or expanded access.

Each request will be carefully considered on a case-by-case basis, as the circumstances and medical histories of all patients are not the same, and local/national laws and regulations vary. Many factors must be weighed when evaluating compassionate use requests including the potential risks and benefits for each patient. Throughout the process, we must also keep the best interests of the entire patient community in mind as we make these important decisions.

Notably, the approval of a compassionate use request is also at the discretion of regulatory agencies in their respective countries. Learn more about the FDA’s expanded access policies and requirements and click here for the European Medicines Agency policy.

If you feel you may be eligible for compassionate use of one of our investigational therapies, your physician must submit a request on your behalf. Treating physicians who would like to request access to one of our investigational therapies are encouraged to contact us at or 877-659-5518. Retrophin anticipates that it will provide an acknowledgement of receipt to the requesting physician within seven business days. Submissions will be reviewed by an internal committee.

The committee will carefully consider each request on a case-by-case basis, based in part on the following:

  • the patient is ineligible to participate in a clinical trial;
  • there is sufficient evidence to expect that the investigational therapy will have an acceptable safety profile for the intended patient;
  • there is adequate supply of the investigational therapy;
  • the patient has been diagnosed with a serious or imminently life-threatening disease or condition;
  • there are no comparable or suitable alternative treatments available or existing; and
  • the requesting physician can meet the explicitly set criteria for treating the intended patient with the investigational therapy.

It’s also important to know that the compassionate use of an investigational therapy may be discontinued at any time at the discretion of the treating physician, the patient or their caregiver, the local regulatory agency, such as the FDA, or Retrophin.

To learn more about our expanded access program and process, please contact or 877-659-5518.