Takeda Pharmaceutical Company Limited’s Ninlaro was granted US FDA approval last November, but conditional approval in Europe was only achieved last week.
In May the European Medicine Agency’s Committee on Human Medicinal Products (CHMP) offered a negative opinion on the small molecule’s efficacy as an orphan drug to treat MM, on the basis that the “benefits of Ninlaro did not outweigh its risks”.
Takeda then requested a re-examination to overturn the decision.
Kate Burd, a Director at Takeda Oncology, told in-Pharmatechnologist.com that following the appeal “[the regulatory] advisory group unanimously agreed to the positive benefit-risk profile of NINLARO, given its efficacy, safety and convenience profile [as an oral drug for multiple myeloma].”
Takeda initially presented data from a trial of 772 adults with MM to propose a restricted approval of Ninlaro for patients which were “harder to treat”.
In response, the committee decided “the data in these subgroups [of harder to treat patients] were not compelling enough and the rationale for assuming greater effectiveness in these patients was not clear.”
Takeda asked for its drug to be reassessed in June, with a positive CHMP opinion being issued on September 15.
According to Burd, “the re-examination procedure did not require or permit submission of new data, but was based upon data from the pivotal Phase III TOURMALINE-MM1 trial.”
Results showed a 35% increase in survival time in adult patients with relapsed/refractory MM compared to those on placebo.
Burd added: “The CHMP Inter-Committee Scientific Advisory Group on Oncology on September 5th gave Takeda the opportunity to present the strength of the TOURMALINE-MM1 data in a face-to-face setting.”
Ninlaro (ixazomib) is an oral proteasome inhibitor, which selectively works to kill myeloma cells in the blood. Originally developed by Millenium Pharmaceuticals, it was acquired by Takeda in 2008.
The drug is prescribed as a second line therapy in combination with generic corticosteroid dexamethasone and Celgene’s chemotherapy drug lenalidomide–trade name Revlimid.
Christophe Bianchi, President of Takeda Oncology, said in a statement: “Ninalaro delivers the proven efficacy of a proteasome inhibitor in a convenient once-weekly pill that can be taken at home.”
Manufacturing of Ninlaro
Kate Burd also told in-Pharmatechnologist that an oral drug for MM was initially considered a challenge to develop:
“The team faced a lot of skepticism as to whether a "peptide" drug could be delivered orally. Nonetheless, our formulations and chemistry teams were able to overcome the challenges and prove it was possible.”
Ninlaro is currently being made in Germany and packaged in Wales, however a new €40m ($43m) facility is being built in Ireland to manufacture the drug.
Takeda announced last week that it is also building a €94m ($100m) manufacturing plant in Germany for its vaccine against Dengue fever – TAK-003.