Multiple myeloma patients in the United Kingdom can now potentially benefit from a new oral pill sold as Ninlaro (ixazomib) after Takeda, the maker of the medicine, struck a deal with the National Health Service, known as NHS England.
The deal authorizes Ninlaro to be distributed through the Cancer Drugs Fund (CDF), a NHS England source of funding for cancer drugs that provides access to promising treatments while further evidence is collected to address clinical uncertainty, among other services.
Under the agreement, Ninlaro will be available under this access program while more evidence is collected on its value.
The deal with NHS England follows a positive draft guidance from the National Institute for Health and Care Excellence (NICE) in December 2017.
So far, studies have shown evidence that Ninlaro is effective at slowing down disease progression compared with standard-of-care, but it is not yet clear if it will help patients live longer.
“We do not yet know the full extent of ixazomib’s benefits to patients, but the early results look promising. Our recommendation means that patients can access this new, oral treatment whilst more data is gathered on whether this treatment will represent value for money for the NHS,” Mirella Marlow, acting director of the Centre for Health Technology Evaluation, said in a NICE press release.
Myeloma is a blood cancer that affects the plasma cells in the bone marrow, which ultimately leads to damage and affects the production of healthy blood cells.
Ninlaro is designed to treat patients who have previously had two or three other treatments. It is given in combination with Revlimid (lenalidomide) and dexamethasone, two compounds used to treat myeloma. It works by slowing down the cancer cell’s ability to digest proteins, and the buildup of excess proteins causes cell death.
“The NICE decision to include ixazomib in the CDF is a real win for the myeloma community. This is a success story of how Takeda has worked together with NICE, NHS England, patient groups and physicians to ensure myeloma patients in England can gain access to this important treatment,” said Dion Warren, vice president and head of oncology business unit in Europe and Canada at Takeda.
In November 2015, the U.S. Food and Drug Administration (FDA) granted the first regulatory global approval for Ninlaro in combination with Revlimid and dexamethasone to treat myeloma patients who had received at least one prior therapy.
However, in the European Union, it took nearly one year for the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) to grant conditional marketing authorization to Ninlaro — also in combination with Revlimid and dexamethasone.
In June, 2016, Takeda received a negative opinion from CHMP recommending against authorizing Ninlaro for myeloma patients, saying the data was not compelling enough.
Those concerns were resolved with data from the Phase 3 TOURMALINE-MM1 clinical trial (NCT01564537), published in April 2016 in The New England Journal of Medicine. The trial found that Ninlaro with Revlimid and dexamethasone resulted in a significant extension in the time a patient lived without disease worsening versus just Revlimid and dexamethasone. The study also reported a favorable benefit-risk profile.
Based on these results, EMA’s CHMP reversed the negative opinion and issued a positive opinion in September, which led to the conditional marketing authorization in November 2016.
Data evaluating how well the treatment works is anticipated to be available by December 2019. This new evidence will be reassessed by NICE to see if the remaining clinical uncertainties have been resolved before Ninlaro can be recommended for routine myeloma treatment.
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