European regulators grant fast path to market for Calliditas’ rare kidney disease drug
European regulators have granted a fast path to market for Calliditas Therapeutics’ potential rare kidney disease drug, Nefecon, weeks after a similar decision by the FDA.
The Swedish biotech said the European Medicines Agency had agreed to a conditional marketing authorisation.
This will allow for the IgA nephropathy (IgAN) drug to be marketed on the basis of mid-stage trial data, with its licence renewed annually until confirmatory data from phase 3 trials is available.
A filing will include an analysis by Calliditas related to the likelihood of achieving success in part B of the ongoing phase 3 study, NeflgArd.
Calliditas noted it already has “strong” phase 2b trial data, a critical statistical analysis, and a solid phase 3 study design that is a larger version of the successful mid-stage trial.
NeflgArd begin in November last year and is still running, with top line data expected in the second half of 2020.
A month ago the FDA said it would accept a readout from NeflgArd after two years, reducing the trial duration from up to six years to around 3.5 years.
The study size has also been cut from 460 to 350 patients for both parts of the trial, cutting costs and recruitment time.
Patients assigned to the placebo arm will also be allowed to cross over on to treatment after the two year pivotal study is completed, generating additional data about the drug’s disease-modifying effect.
Nefecon is a formulation of the established immunology drug budesonide that dissolves only when it reaches the ileum – the last part of the intestine.
This stops organs in the ileum wall known as Peyer’s patches from producing faulty IgA antibodies which are thought to cause the disease.
Although classified as an orphan disease, the company estimates that there are about 130,000-150,000 people in the US and about 200,000 people in Europe with the condition.
This comparatively large patient population could translate into sales in excess of $1 billion annually if approved by regulators, according to the company’s estimates, and there are no specially approved competitors.
Don't miss your daily pharmaphorum news.
SUBSCRIBE free here.