Canadian biopharmaceutical firm Neurochem said yesterday that the US Food and Drug Administration has issued an approvable letter regarding its developmental amyloid A amyloidosis treatment Kiacta.
The letter requests additional safety and efficacy data on Kiacta (eprodisate), which the agency said would need to be produced either through another clinical trial or additional follow-up of patients in the existing study.
However, Neurochem is confident that it can satisfy the agency’s requirements without another trial, a bullish stance that helped its share price advance 22% yesterday to close at C$13 on the Toronto Stock Exchange. It also aims to file for approval of the product in Europe in September.
Kiacta is partnered with Johnson & Johnson subsidiary Centocor, which has exclusive worldwide distribution rights to the product with the exception of Canada, Switzerland, Japan, China, South Korea and Taiwan.
The FDA also requested further manufacturing and pharmacokinetic information and said that a QT clinical study should be carried out as a post-marketing commitment.
Neurochem's president, Francesco Bellini, said that it would work with the FDA to obtain the requested data, without the need for a further trial.
A progressive and fatal condition, AA amyloidosis occurs in a proportion of patients with chronic inflammatory disorders, chronic infections and inherited diseases. The kidney is the organ most frequently affected and progression to dialysis and end stage renal disease is the most common cause of death in this disease.
Currently, there is no FDA-approved therapy to treat AA amyloidosis and half of all patients diagnosed with the disease die within five years of diagnosis.