Novartis Drug Safety Concerns Reported

02 June 2016

Safety concerns have prompted the pharmaceutical company Novartis to pause enrolment in the Phase 2 clinical trial of their SMN2 splice-modifying drug LMI070.

The Phase 2 trial, which is being conducted in SMA patients aged 6 months or less (click here for more information), has been halted due to evidence from animal safety experiments being performed in conjunction with the patient trial. Animals treated with LMI070 every day for a year have shown unexpected complications with their blood vessels, kidneys, spinal cord and peripheral nerves, such as the motor neurons.

Unlike the animals, patients in the trial are taking weekly doses of the drug, and it is perhaps for this reason that the trial is being continued in those patients already receiving LMI070 treatment. Those patients already enrolled are to be very carefully monitored and have the option to leave the trial if they wish.

Reported on the Cure SMA website, this news marks the second such safety concern over a drug being tested in SMA patients (click here for more information), and highlights the importance of performing detailed and thorough analyses of experimental therapies before administering them on a large scale to SMA patients.

Novartis are reported to be working closely with health authorities to determine the fate of LMI070.


Further Information

Cure SMA article on the safety concerns

LMI070 clinical trial page



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