Japanese Orphan designation for Takeda's ixazomib for relapsed/refractory multiple myeloma

The Japanese Ministry of Health, Labor and Welfare has granted Orphan Drug designation to drug major Takeda Pharmaceutical’s (TYO: 4502) ixazomib (MLN9708), an investigational oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma.

The Orphan Drug designation is a system for supporting and promoting the development of drugs that are not sufficiently researched and developed due to a small number of patients, regardless of high medical need.

Ixazomib's Phase III trials for patients with multiple myeloma are now being conducted in Japan. The drug became available in the USA, under the brand name Ninlaro, in December 2015 after the US Food and Drug Administration approval as a treatment for patients with relapsed and/or refractory multiple myeloma who have received at least one prior therapy. The drug is currently under review in the European Union. The European Medicines Agency granted ixazomib an accelerated assessment in July 2015 and accepted its Marketing Authorization Application in August 2015 for the treatment of patients with relapsed and/or refractory multiple myeloma.

The global treatment market for multiple myeloma will rise in value from $8.9 billion in 2014 to an estimated $22.4 billion by 2023, representing an impressive compound annual growth rate (CAGR) of 11.2%, GlobalData research indicates.