Cambridge, MA and Osaka, Japan (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.

About Multiple Myeloma

Multiple myeloma is a cancer of the plasma cells, which are found in the bone marrow. In multiple myeloma, a group of plasma cells, or myeloma cells, becomes can­cer­ous and multiplies, in­creas­ing the number of plasma cells to a higher than normal level. Because plasma cells circulate widely in the body, they have the poten­tial to affect many bones in the body, possibly resulting in compression fractures, lytic bone lesions and related pain. Multiple myeloma can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count, with some of the more common symp­toms in­­clud­ing bone pain and fatigue, a symp­tom of anemia. Multiple myeloma is a rare form of cancer, with approx­i­mately 20,000 new cases in the U.S. and 114,000 new cases globally per year.

About Ixazomib

Ixazomib is an inves­ti­ga­tional oral pro­te­a­some inhibitor which is being studied in multiple myeloma, systemic light-chain (AL) amyloidosis, and other malig­nan­cies. Ixazomib was granted orphan drug desig­na­tion in multiple myeloma in both the U.S. and Europe in 2011 and for AL amy­loid­osis in both the U.S. and Europe in 2012. Ixazomib received Break­through Therapy status by the U.S. FDA for re­lapsed or refractory AL amy­loid­osis in 2014. It is also the first oral pro­te­a­some inhibitor to enter Phase 3 clin­i­cal trials.

Ixazomib’s clin­i­cal devel­op­ment pro­gram further reinforces Takeda’s ongoing commitment to devel­op­ing inno­va­tive ther­a­pies for people living with multiple myeloma world­wide and the health­care professionals who treat them. Five global Phase 3 trials are ongoing:

• TOURMALINE-MM1, investigating ixazomib vs. placebo, in combination with lenalidomide and dexa­metha­sone in relapsed and/or refractory multiple myeloma
• TOURMALINE-MM2, investigating ixazomib vs. placebo, in combination with lenalidomide and dexa­metha­sone in patients with newly diagnosed multiple myeloma
• TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma following induction therapy and autologous stem cell transplant (ASCT)
• TOURMALINE-MM4, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma who have not undergone ASCT
• TOURMALINE-AL1, investigating ixazomib plus dexa­metha­sone vs. physician choice of selected reg­i­mens in patients with relapsed or refractory AL amyloidosis
• For additional information on the ongoing Phase 3 studies please visit www.clinicaltrials.gov.

About Takeda Oncology

Takeda Oncology is the brand for the global on­col­ogy business unit of Takeda Pharma­ceu­tical Company Limited. Takeda aspires to cure cancer by delivering novel medicines to meet the unique and urgent needs of people living with cancer, their loved ones and the health care providers who sup­port them around the world. Takeda is the 11th largest global on­col­ogy com­pany with a portfolio of paradigm-changing ther­a­pies and an extensive pipe­line of inves­ti­ga­tional prod­ucts that have the poten­tial to sig­nif­i­cantly im­prove patient out­comes across a number of cancers. By combining the power of leading scientific minds with our agile and entrepreneurial spirit and the vast resources of a global pharma­ceu­tical com­pany, Takeda Oncology is find­ing new and inno­va­tive ways to im­prove the treat­ment of cancer. Additional in­­for­ma­tion about Takeda Oncology is avail­able through its website, www.takedaoncology.com.

About Takeda

Located in Osaka, Japan, Takeda (TSE: 4502) is a research-based global com­pany with its main focus on pharma­ceu­ticals. As the largest pharma­ceu­tical com­pany in Japan and one of the global leaders of the in­dus­try, Takeda is committed to strive to­wards better health for people world­wide through leading inno­va­t in medicine.

Additional in­­for­ma­tion about Takeda is avail­able through its corporate website, www.takeda.com.

Source: Takeda.