Bitopertin Expanded Access Program

Disc Medicine’s Expanded Access Program allows eligible people with EPP or XLP to be considered for access to bitopertin, an investigational medicine being studied for these conditions, outside of a clinical trial.

Four sites for Disc Medicine’s Expanded Access Program (EAP), for bitopertin are now open.

Some important things to know about the EAP.

• The EAP is currently open to residents of the United States for people with EPP or XLP ages 12 and older. Patients will need to meet specific criteria to be considered. More information about the criteria is available at ClinicalTrials.gov.

• Each EAP site may have a different process for reviewing interested patients, scheduling visits, and starting treatment. 

• In general, patients should expect one to two visits to receive medication, followed by a visit at approximately three months and then follow-up visits about every six months to complete assessments. 

• Participating sites will have different insurance requirements. Insurance may be needed to cover clinic visits, laboratory work, or other care-related costs. The medication itself is provided through the EAP. 

• Patients may be able to travel to participate at an EAP site, but travel and lodging expenses are generally the responsibility of the patient or family.

Accessing the EAP

Four sites for Disc Medicine’s Expanded Access Program (EAP) for bitopertin are now open with more expected in the coming months. Open sites are:

• Boston, MA | Mark Amster, MD 

• New York, NY | Manisha Balwani, MD

• Miami, FL | Cynthia Levy, MD

• Winston-Salem, NC | Sean Rudnick, MD

United Porphyrias Association helps connect interested patients with available sites. As new sites become active, UPA will reach out directly to interested patient, especially when a site opens in or near their region. 

Please complete the interest form for updates and to be contacted when a site becomes available in your region.

Please note that completing the form does not mean you are enrolled in the program or guaranteed access to bitopertin.

Bitopertin is an investigational medicine. It is not approved by any regulatory authority, and its safety and effectiveness have not been established.

We will share more as information becomes available.