The battle to get an oral treatment for multiple sclerosis onto the market is hotting up, after Novartis confirmed it has filed fingolimod with regulators in the USA and Europe.
The Swiss major says it submitted applications for the pill, also known as FTY720, to the US Food and Drug Administration and the European Medicines Agency last month at the lower 0.5mg dose as the results from the studies indicate that this has the most positive benefit-risk profile to the 1.25mg dose.
The filing comes a couple of months after Merck KGaA’ received a ‘refuse to file’ letter from the US agency for cladribine, which the German firm hopes will be the first oral treatment to be approved for relapsing forms of MS. The latter was submitted to the EMEA last summer and Merck hopes to meet with the FDA shortly to iron out the problems of its sunbmission across the Atlantic.
News of the Novartis filing came just as data from trials for both fingolimod and cladribine were published in New England Journal of Medicine. First up, in two late-stage trials of FTY720, the drug proved to be effective in reducing relapses, disability progression and MRI lesions in MS.
In the one-year TRANSFORMS study involved 1,292 patients with relapsing-remitting MS who were randomised to receive one of the two aforementioned doses of fingolimod daily, or Biogen Idec's blockbuster injectable Avonex (interferon beta-1a) once a week. Data showed that the annualised relapse rate was significantly lower in both fingolimod groups and the lower 0.5mg dose reduced relapses by 52% compared with Avonex.
Also full results from the two-year 1,272-patient FREEDOMS trial of fingolimod, revealed that patients taking the lower and higher doses of fingolimod had a 54% and 60% reduction, respectively, in the annualised relapse rate, compared with placebo.
The NEJM also published previously-released Phase III data on cladribine, namely the two-year, 1,326-patient CLARITY study. These also showed that patients with relapsing-remitting MS who took either of two dosages of Merck’s drug had a significantly lower annualised relapse rate, compared with those who received placebo.
In an editorial in the NEJM, neurologist William Carroll said that “the long-awaited arrival of oral formulations for the treatment of relapsing-remitting MS is welcome news for the estimated 2.5 million people worldwide who have this chronic, disabling disease. He added that the fingolimod and cladribine studies “provide a new horizon for patients” and “a welcome increase in the range of treatment options".
Dr Carroll cautioned that “time will determine the long-term effectiveness of these treatments in delaying the development of irreversible disability”, but as “ongoing, real-life experiments they will contribute to our understanding of this enigmatic disease.”
Doug Brown, biomedical research manager at the UK’s MS Society, said: “this is great news…and signifies a shifting tide in the treatment of the condition. He added that “availability of oral therapies will give people greater choice and being able to take a tablet instead of unpleasant injections will come as welcome relief”.