Multiple Myeloma Drug Granted Conditional Approval in Europe

Ninlaro regimen increased progression-free survival by 40% in patients with multiple myeloma.

Takeda Pharmaceutical Company announced that the European Commission has granted conditional approval of Ninlaro (izazomib), the first oral proteasome inhibitor to be approved for multiple myeloma.

The capsules are indicated in combination with lenalidomide and dexamethasone for treatment-experienced patients with multiple myeloma, according to a press release from Takeda. This approval follows positive opinion from the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use.

“For myeloma patients living in Europe, the approval of Ninlaro means we have a new and effective treatment option available when we relapse,” said Bob Munro, a patient with multiple myeloma from the United Kingdom. “I applaud the European Commission for recognizing the additional benefit that Ninlaro will bring to patients, who not only want treatment options that are effective and tolerable, but also appreciate the convenient option of taking an oral treatment. I strongly hope this will be made available by national health systems across Europe as soon as possible.”

The approval was based on positive data from the phase 3 TOURMALINE-MM1 clinical trial, which showed that Ninlaro plus lenalidomide and dexamethasone increased progression-free survival by 40% (6 months) in patients with relapsed and refractory multiple myeloma, compared with placebo, lenalidomide, or dexamethasone, Takeda reported.

Progression-free survival was also demonstrated in pre-specified subgroups of patients taking the Ninlaro regimen. Takeda is planning to conduct multiple follow-up analyses in 2017 to determine the impact the regimen has on overall survival.

The TOURMALINE clinical development program includes 5 ongoing clinical trials, including 4 for multiple myeloma. The development program reinforces Takeda’s commitment to creating novel treatments for multiple myeloma, according to the press release.

“With the approval of Ninlaro by the European Commission, physicians across the region will have the option to prescribe an all-oral triplet regimen to treat patients with multiple myeloma who have received at least 1 prior therapy,” said Philippe Moreau, MD, head of the Hematology Department at the University Hospital of Nantes, France. “In the TOURMALINE-MM1 study, we saw a clinically meaningful 6-month improvement in progression-free survival with Ninlaro, evidence that has supported its approval in Europe. As a hematologist, I welcome the availability of this treatment to address a devastating disease like multiple myeloma.”

Ninlaro is now approved in all countries in the European Economic Area, which includes the European Union, Norway, Lichtenstein, and Iceland, Takeda reported. The drug is also licensed to be marketed in the United States, Canada, Israel, Australia, and Venezuela.

It received approval from the FDA in 2015 after it was granted orphan drug designation in 2011. Ninlaro also was granted orphan drug designation and Breakthrough Therapy status by the FDA for the treatment of patients with relapsed or refractory systemic light-chain amyloidosis.

Takeda has already submitted marketing authorization applications to numerous regulatory authorities worldwide in hopes of additional approvals for multiple myeloma.

“When developing Ninlaro, Takeda Oncology’s scientists sought to formulate an efficacious and unique oral proteasome inhibitor with a manageable safety profile. Ninlaro delivers the proven efficacy of a proteasome inhibitor in a convenient once-weekly pill that can be taken at home,” said Christophe Bianchi, MD, president of Takeda Oncology. “Ninlaro has the potential to help European patients with relapsed multiple myeloma by removing some of the barriers that can stand in the way of optimal treatment. With Ninlaro, our hope is that many patients will be able to continue therapy until disease progression. Following the European Commission’s approval, we will continue to study Ninlaro in a variety of settings in the hopes that we can bring this medicine to as many of the patients who may benefit from it as possible.”