Expanded Access Program

In This Section

Expanded access, sometimes called "compassionate use," is the use of an investigational medical product outside of a clinical trial (i.e., one that has not yet been approved). At Novelion Therapeutics, and our subsidiary Aegerion Pharmaceuticals, we are committed to increasing awareness of and knowledge about our expanded access programs and the procedures for obtaining access to human investigational drugs.

We are keenly aware that patients living with rare diseases, their families and caregivers frequently find themselves surrounded by a healthcare system that does not understand their health condition or have the resources to help them. Our aim is to be a catalyst of change, easing the burden on these patients by educating the community, developing transformative therapies and offering comprehensive support.

With that as our goal, we work to develop medicines and provide access to patients globally as quickly and safely as possible. This includes working with various regulatory agencies to design and conduct clinical trials, submit relevant data from those studies to the agencies, and seek formal approval for these medicines in the targeted indications.

We also recognize that there may be cases where medicines are urgently needed and alternatives for treatment are either absent or have been exhausted. In this case, Novelion Therapeutics, and our subsidiary Aegerion Pharmaceuticals, have an Expanded Access Program (EAP) that may be appropriate for a patient who does not yet have access to a therapy that has shown favorable benefit/risk in clinical trials.

An EAP is designed to provide a patient with access to a medicine before it is approved by a national health and/or reimbursement authority and outside of the clinical trial setting.

We are committed to providing transformative therapies and offering best-in-class support for patients who might benefit from our therapies and will facilitate access to the best of our ability. Please find the criteria and links below to begin the process of determining eligibility for the Expanded Access Program. Only physicians may enroll patients in a clinical trial or EAP.

  • We are committed to helping provide access to our therapies to patients in certain places where the drug is not currently approved.  
  • We evaluate requests to determine that the provision of drug will in no way intervene with ongoing clinical trials, regulatory approval or future reimbursement.
  • We support patients who have a serious or life-threatening disease where no other treatment options are available.
  • We ensure that the provision of drug in no ways compromises the supply to patients currently on therapy.
  • We abide by all local, regional, national and global regulations and laws when permitting access for individual patients.
  • We never implement access to unapproved medicines in any country in a way that could be construed as marketing or promotion of a product prior to regulatory approval.

Lomitapide Expanded Access Program (Aegerion Pharmaceuticals) 

The program has certain clinical eligibility criteria.  Pediatric patients with HoFH may be eligible for access to lomitapide through the expanded access program.

Metreleptin Expanded Access Program (Aegerion Pharmaceuticals)

The program has certain clinical eligibility criteria.  Patients with lipodystrophy and congenital leptin deficiency may be eligible for access to metreleptin through the expanded access program.  Please note the Metreleptin Expanded Access Program is provided through our partnership with the Clinigen Group.

Please note:  Applications must be submitted by the HealthCare Professional. Patient eligibility will be determined by Novelion/Aegerion in accordance with established policies and procedures. The acceptance and processing of this application does not guarantee that access to investigational product will be provided.