Pre-Approval Access Policy
We are conducting clinical studies of an investigational medicine, known as apraglutide, in two serious, rare disorders: Short Bowel Syndrome (SBS) in patients who are dependent on parenteral support and acute Graft Versus Host Disease (GVHD) involving the GI tract, that is failing to respond sufficiently to steroids.
- In SBS with intestinal failure, patients have insufficient working intestine to meet their nutritional and/or hydration needs and rely on intravenous (IV) nutrition and/or IV hydration. Our pivotal Phase III study has completed enrollment.
- Acute GVHD (i.e., tends to start within 100 days of an allogeneic stem cell transplant) can impact the GI tract. In around 50% of cases, acute GVHD of the GI tract fails to respond sufficiently to steroids, which can worsen a patient’s prognosis. A Phase II proof-of-concept study has completed enrollment in this patient population.
We remain committed to making apraglutide available as quickly as we can to appropriate patients, following regulatory authority approval, which depends in part on positive data from our clinical studies and the evaluation of these data by regulatory and reimbursement authorities.
Pre-approval access, sometimes known as Expanded Access or Compassionate Use, refers to the provision of an investigational medicine outside of a clinical trial and ahead of regulatory or reimbursement body approval.
At this current time, we believe that participating in clinical studies is the safest way to access our new investigational medicine, under the close monitoring of a specialist clinical research team. Therefore, we do not provide access to apraglutide through Pre-approval Access programs, or outside of clinical studies more generally. This policy may be reviewed and amended in the future.
Details about our clinical studies, including eligibility requirements for participating in ongoing or planned studies are published in public databases such as www.clinicaltrials.gov and www.clinicaltrialsregister.eu.
If you have further questions, please speak with your healthcare provider or contact our Medical Information Team at [email protected].