Regulatory Decision Summary for HepaBridge

Decision issued
No decision was issued by Health Canada. The company cancelled its submission before a final decision was issued.

Date of cancellation
2018/07/11

What was the purpose of this submission?
The intent of this New Drug Submission was to seek market authorisation of HepaBridge for the proposed indication: the treatment of pediatric patients from birth to less than 3 years of age suffering from severe urea cycle disorders for whom orthotopic liver transplantation is considered a treatment option at a later time point.

The intent of the HHLivC therapy was to substitute the urea cycle function until the children are old enough and have reached the appropriate body weight to receive a liver transplant.

What did the company submit to supports its submission?
The sponsor submitted drug quality, pre-clinical and clinical components. The pivotal clinical information consisted of data from 22 patients enrolled in two studies (CCD02 and CCD05). Both of them were phase II studies to assess the safety and efficacy of liver cell infusion with HHLivC in children with urea cycle defects (UCD) between the ages of 1 day to 5 years:

• CCD02 was an open, prospective, uncontrolled, multi-centre study, with safety as the primary endpoint. 12 patients have been enrolled and treated as planned per protocol.
• CCD05 was an open, prospective, multi-centre study, with the primary efficacy endpoint being 13C-ureagenesis activity. This study was intended to include 21 patients, however, due to low recruitment rate, it was terminated after 10 patients were enrolled, of which 9 were treated with HHLivC.

The treatment comprises catheter placement, liver cell administration (HHLivC, 6 daily doses, with body weight adjusted dose), long-term concomitant immunosuppressive treatment and standard of care.

Phase 3 trial data were not part of the submission.

What was the status of the submission when it was cancelled? What was Health Canadas assessment of the submission at the time of cancellation?
At the time of the cancellation, the review of the submission was nearing completion. Health Canada had identified some deficiencies in the data that would have precluded issuing an approval. The sponsor chose to cancel their submission without prejudice to refiling at a later date.

What consequences does the cancellation have for patients accessing the drug under the Special Access Programme (SAP), or via clinical trials?
There is no expected impact for patients using SAP or in clinical trials.

Requests for special access to HepaBridge will continue to be considered on a case-by-case basis. For more information about the Special Access Programme refer to the programmes web site: www.healthcanada.gc.ca/sap or www.santecanada.gc.ca/pas

Additional information

Proposed Brand Name:
HepaBridge

Manufacturer
Promethera Biosciences S.A./N.V

Drug Identification Numbers issued
N/A

Date filed
2017/06/16