Cambridge, Mass. and Osaka, Japan, May 27, 2016 – Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion, recommending against the authorization of NINLARO® (ixazomib) capsules, an oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma. Takeda intends to appeal this opinion and request a re-examination by the CHMP.
“We are disappointed by the CHMP’s opinion. With the support of European key medical experts, we will continue our efforts working closely with the CHMP to make NINLARO – the first oral proteasome inhibitor – available for patients in Europe,” said Christophe Bianchi, M.D., President, Takeda Oncology. “Despite recent progress, myeloma remains an intractable disease, and patients suffering from multiple myeloma and their treating physicians need more options to improve outcomes. We stand behind the TOURMALINE-MM1 trial data, which were recently published in the New England Journal of Medicine and demonstrated a significant extension in progression-free survival for NINLARO + lenalidomide and dexamethasone vs. placebo + lenalidomide and dexamethasone and a favorable benefit-risk profile.”
“After years of treating patients, I have yet to see two people whose diseases are exactly alike. The diversity of patients with multiple myeloma demands a wide range of innovative treatment options that offer efficacy, tolerable safety profiles and convenience, which are especially important benefits for elderly populations,” said Philippe Moreau, M.D., University of Nantes, France. “In Europe, where no oral proteasome inhibitor is available, NINLARO would fill a noticeable void and enable the first all-oral triplet combination therapy for patients with relapsed or refractory multiple myeloma.”
NINLARO was approved by the U.S. Food and Drug Administration (FDA) in November 2015 following a priority review. In the U.S., NINLARO is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. The FDA approval of NINLARO marked the first global regulatory approval of ixazomib. Takeda also has submitted applications for approval of ixazomib to additional regulatory authorities around the world. In addition to the TOURMALINE-MM1 trial that is forming the basis of these global regulatory submissions in relapsed and refractory multiple myeloma, ixazomib is being investigated in a number of other multiple myeloma treatment settings.
There will be no significant impact to Takeda’s fiscal year 2016 financials due to the CHMP opinion.
About NINLARO® (ixazomib)
NINLARO®(ixazomib) is an investigational oral proteasome inhibitor which is being studied in multiple myeloma and systemic light-chain (AL) amyloidosis. It was the first oral proteasome inhibitor to enter Phase 3 clinical trials and to receive approval.
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 systemic light-chain (AL) amyloidosis, a related ultra orphan disease, in 2014.
The comprehensive ixazomib clinical development program, TOURMALINE, further reinforces Takeda’s ongoing commitment to developing innovative therapies for people living with multiple myeloma worldwide and the healthcare professionals who treat them. TOURMALINE includes a total of five ongoing pivotal trials – four investigating every major multiple myeloma patient population and one in light-chain amyloidosis:
In addition to the TOURMALINE program, a large number of investigator initiated studies are evaluating ixazomib for patients globally.
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 monoclonal plasma cells, or myeloma cells, becomes cancerous and multiplies. These malignant plasma cells 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 39,000 new cases in the EU and 114,000 new cases globally per year.
Important Safety Information (U.S.)
WARNINGS AND PRECAUTIONS
The most common adverse reactions (≥ 20%) in the NINLARO regimen and greater than the placebo regimen, respectively, were diarrhea (42%, 36%), constipation (34%, 25%), thrombocytopenia (78%, 54%; pooled from adverse events and laboratory data), peripheral neuropathy (28%, 21%), nausea (26%, 21%), peripheral edema (25%, 18%), vomiting (22%, 11%), and back pain (21%, 16%). Serious adverse reactions reported in ≥ 2% of patients included thrombocytopenia (2%) and diarrhea (2%).
DRUG INTERACTIONS: Avoid concomitant administration of NINLARO with strong CYP3A inducers.
Please see NINLARO full U.S. Prescribing Information: https://www.ninlarohcp.com/safety.
About Takeda Pharmaceutical Company
Takeda Pharmaceutical Company Limited is a global, R&D-driven pharmaceutical company committed to bringing better health and a brighter future to patients by translating science into life-changing medicines. Takeda focuses its research efforts on oncology, gastroenterology and central nervous system therapeutic areas. It also has specific development programs in specialty cardiovascular diseases as well as late-stage candidates for vaccines. Takeda conducts R&D both internally and with partners to stay at the leading edge of innovation. New innovative products, especially in oncology and gastroenterology, as well as its presence in emerging markets, fuel the growth of Takeda. More than 30,000 Takeda employees are committed to improving quality of life for patients, working with our partners in health care in more than 70 countries. For more information, visit http://www.takeda.com/news.
Additional information about Takeda is available through its corporate website, www.takeda.com, and additional information about Takeda Oncology, the brand for the global oncology business unit of Takeda Pharmaceutical Company Limited, is available through its website, www.takedaoncology.com.
+44 7974 151510
Media outside Japan/EU
+81 (0) 3-3278-2417