Novartis has withdrawn the EMA filing for its drug canakinumab as a potential cardiovascular treatment after the EU regulator followed its US counterpart in raising doubts about its data.
The decision is a further setback for this expansion in the use of canakinumab, which Novartis already markets as an inflammatory disease treatment under the brand name Ilaris.
The Swiss pharma giant had touted the drug as a future blockbuster in the cardiovascular setting, and suggesting it could transform treatment of millions of patients vulnerable to further heart attacks and strokes.
The company has completed a phase 3 study of canakinumab as a treatment to prevent stroke and myocardial infarction (MI) or death in patients who have already suffered a heart attack – but has now opted for a tactical retreat in the face of resistance from regulators.
The EMA filing withdrawal news followed a similar negative sentiment from the FDA, which issued a complete response letter to a filing seeking approval of the drug in October for cardiovascular risk reduction.
On Friday the EMA said its Committee for Medicinal Products for Human Use (CHMP) had “concerns” over the application, and was minded to issue a negative opinion on the filing, and said the data submitted by Novartis was “not robust enough” to show the drug’s efficacy in all patients who have had an MI.
It noted that the drug had only demonstrated “modest” benefits in the data, particularly in patients who also took statins. The EU regulator also said that the benefits of the drug were not considered enough to outweigh the increased risks of serious infections in patients treated with the medicine, and questioned the ‘appropriateness’ of methods chosen for selecting patients and of monitoring the effectiveness of the treatment.
The EMA indicated that Novartis had withdrawn its application after concluding that it would not be possible to address the concerns within the agreed timeframe.
The EU regulator made it clear that studies of canakinumab for the treatment of other conditions would be unaffected by the decision.
This is a markedly different picture to when Novartis reported encouraging data from its phase 3 CANTOS trial in 2017. This showed that injections of canakinumab every three months reduced the risk of major adverse cardiovascular events in certain patients who had a prior heart attack by 15% and has met its primary endpoint.
The company has previously suggested that some 4m people in the world’s largest markets could benefit from the drug, and analysts had previously predicted peak annual revenues in excess of $2bn.
While it still looks likely that Novartis will pursue the drug’s use in this setting, the path forward – potentially including the need for further large scale trials – remains unclear, and lingering reservations about safety and efficacy will temper its commercial potential.