Regulatory Decision Summary for Panobinostat (*Farydak)

 

Decision issued

A Notice of Deficiency was issued by Health Canada. The company cancelled its submission before a final decision was issued.

Date of cancellation

2016-06-23

 

What was the purpose of this submission?

A New Drug Submission (NDS) was filed to obtain market authorization for panobinostat, a histone deacetylase (HDAC) inhibitor, to be used in combination with bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received bortezomib and an immunomodulatory agent.

What did the company submit to support its submission?

The submission included a clinical and non-clinical package, a risk management plan, and the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) reviews.

The panobinostat clinical development program in multiple myeloma focused on panobinostat in combination with bortezomib and dexamethasone, and includes a Phase III study, one supportive Phase II study, and safety and preliminary efficacy data from the dose expansion phase of a Phase Ib study. The Phase III study was a randomized, double-blind, placebo controlled study in patients with relapsed or relapsed and refractory multiple myeloma who had received one to three prior lines of therapy, and who were not refractory to prior bortezomib treatment. In the control arm, placebo was given on top of standard of care (bortezomib and dexamethasone). Of important note, the indication for use of panobinostat proposed by Novartis is not in line with the overall Phase III study population.

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 not complete; however Health Canada had identified a major deficiency in the information provided that precluded continuation of the review. Specifically, there was a significant gap in the benefit/risk assessment provided in the NDS. The benefit/risk assessment had made a positive conclusion for the overall study group (that is, patients with relapsed and refractory multiple myeloma who have received one to three prior lines of therapy), but did not explain why the proposed indication was restricted to patients with multiple myeloma who have received bortezomib and an immunomodulatory agent. As a result, Health Canada issued a Notice of Deficiency outlining this major objection in addition to a number of other concerns identified during the review.

Following issuance of the Notice of Deficiency, Novartis withdrew the submission for panobinostat for the treatment of patients with multiple myeloma. This withdrawal was without prejudice to a resubmission when data from new studies will be available.

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 panobinostat 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: http://www.healthcanada.gc.ca/sap.

There are two approved clinical trials underway in Canada for the use of panobinostat in the treatment of multiple myeloma. No impact is expected for patients participating in these studies.

Additional information

*Proposed Brand Name
Farydak