Novartis (NVS) is reporting that one of its shining star drugs in development may be in trouble. Doctors in the trial for FTY720, also known as Fingolimod, have reported a case of hemorrhaging focal encephalitis in one patient. The company said the patient concerned was recruited into the study seven months before hospital admission for the reported event, before which no new disease activity had occurred.

Eric Althoff from Novartis commented on the scenario:

“As per normal procedures, this particular case was communicated in a timely fashion to health authorities and study investigators. It is difficult to interpret an isolated case report without the benefit of the full Phase III data set. At this stage, a relationship with FTY720 can neither be excluded nor confirmed.”

FTY720 works as an immunosuppressant, causing lymphopenia by preventing lymphocytes from leaving lymph nodes. The drug has already demonstrated superiority over Biogen’s Avonex and is currently in an ex-US Phase 3 study called FREEDOMS I. The trial is comparing two doses of the drug vs placebo in 1,272 MS patients.

The drug is a potential star because it is given orally (all MS drugs on the market are injection) and has already demonstrated superior efficacy over Avonex. The main concern is safety, safety and safety. Up until now, the known safety issues had been transient heart rate reductions, elevated liver enzymes, skin cancer, infections (there have already been two fatal viral infections) and minor increases in blood pressure. This is the first serious brain inflammation case in the trial which would put concerns about the drug’s safety right up there with Tysabri.

The safety issue might be a positive for Merck’s cladribine which is in a race with NVS to become the first oral MS therapy. Cladribine also had some issues earlier this year when the company reported four patients had died of cancer.

Stay tuned…

Image from bw

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