[Beacon Staff]

Ixazomib gains FDA priority review

by Beacon Staff on Wed Sep 09, 2015 8:54 am

Once again, we have more myeloma-related regulatory news this morning. Takeda, the company that is developing ixazomib as a new treatment for multiple myeloma, has announced that the drug has been granted priority review by the U.S. Food and Drug Administration (FDA).

Here is a link to the Takeda press release announcing the news. And here are the first few paragraphs from the press release:

Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that the U.S. Food and Drug Administration (FDA) has granted Priority Review status to the New Drug Application (NDA) for ixazomib, the first investigational oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma.

“We are encouraged that both the U.S. and European regulatory bodies have de­termined that the ixazomib applications qualify for an expedited review, under­scor­ing the importance of new treatment options for patients with relapsed / refractory multiple myeloma,” said Melody Brown, Vice President of Regulatory Affairs, Takeda. “Our ixazomib program is designed to evaluate whether sustained therapy with an oral proteasome inhibitor improves the outcomes of patients living with multiple myeloma. There is a significant unmet medical need in multiple myeloma and we look forward to working with the regulatory bodies to bring ixazomib to patients.”

The FDA may grant Priority Review status, which includes expedited review, to the evaluation of applications for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or efficacy over existing treatment. Ixazomib was recently granted accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

The NDA submission for ixazomib was primarily based on the results of the first pre-specified interim analysis of the pivotal Phase 3 trial, TOURMALINE-MM1. This study is an international, randomized, double-blind, placebo controlled clinical trial of 722 patients designed to evaluate the superiority of ixazomib plus lena­lido­mide and dexamethasone over placebo plus lenalidomide and dexamethasone in adult patients with relapsed and/or refractory multiple myeloma. Patients continue to be treated to progression in this trial and will be evaluated for long-term outcomes.

[Robert]

Re: Ixazomib gains FDA priority review

by Robert on Thu Sep 10, 2015 9:53 am

This is great news. Thank you for posting. Has any of the research shown that ixazomib is as effective as bortezomib (Velcade) for high risk cytogenetics?

[Cheryl G]

Re: Ixazomib gains FDA priority review

by Cheryl G on Thu Sep 10, 2015 11:49 am

To be honest, I'm a little surprised that ixazomib got a priority review. Kyprolis, for example, did not get a priority review, and neither did Pomalyst. Both, like ixazomib, were additional drugs in existing classes of myeloma treatments (ixazomib and Kyprolis = new proteasome inhibitors, like Velcade; Pomalyst = third immunodulatory agent, after thalidomide and Revlimid).

I guess the fact that ixazomib is an oral proteasome inhibitor made a difference to the FDA, leading it to grant priority review.

Birdwatcher – There aren't many published results of trials that have tested ixazomib in myeloma patients, and the results that are available don't give much information about how the drug may do in high-risk patients. There is a breakdown in this published paper,

SK Kumar, "Phase 2 trial of ixazomib in patients with relapsed multiple myeloma not refractory to bortezomib," Blood Cancer Journal, Aug 2015 (full text of article)

but as you'll see in this table, only 4 patients in the trial were classified as having high-risk disease base on the MSmart criteria.

[Robert]

Re: Ixazomib gains FDA priority review

by Robert on Thu Sep 10, 2015 12:06 pm

Cheryl,

Thank you for the links to the journal article. Looks like it would be wise for those of us with high-risk features to continue watching the research.