Raptor’s orphan drug Procysbi has been given the green light in the EU, having received marketing authorisation from the European Commission last Thursday. Procysbi, which comprises gastro-resistant capsules of cysteamine, is an orphan medical product for the treatment of nephropathic cystinosis, a rare and life-threatening metabolic lysosomal storage disorder that can be fatal by age 10 if left untreated.
Nephropathic cystinosis accounts for about 95% of diagnosed cases of cystinosis, which affects about 800 patients in the EU and 2,000 worldwide. The toxic accumulation of cystine through all tissues and organs in the body leads to progressive tissue damage and multi-organ failure, including kidney failure, blindness and muscle wasting.
The condition usually requires lifelong treatment with cystine depletion from infancy, but poor patient compliance to therapy has remained a major obstacle in controlling cystine levels. While Procysbi has the same ingredient as the current therapy, Mylan’s Cystagon, it can be taken every 12 hours rather than every 6 hours. The difference between the two therapies may seem subtle, but Raptor says that Procysbi’s delayed release properties means the drug is more tolerable than the current offering – and therefore patients may be less likely to skip doses. Considering that even a brief interruption to daily therapy can expose tissues to toxic cystine accumulation, Dr Starr says that Procysbi’s advantages “add up to a significant benefit”.
The drug was approved in the EU off the back of data from six clinical trials, including a Phase III trial involving 43 patients with nephropathic cystinosis, and was approved in the US by the FDA last April, where it costs $250,000 on average.
Raptor’s CEO, Christopher Starr, said that “Active dialogue has begun with a number of EU member states initially as part of a phased introduction of PROCYSBI to patients with this debilitating disease.”
Read the press release >
The World Orphan Drug Congress Europe brings together pioneers from the orphan drug community, including key opinion leaders from the payer, HTA, public health and patient advocacy bodies. Download the brochure for the World Orphan Drug Congress Europe 2013, 14-15 November 2013, Geneva.