Neurochem Inc announced that Fibrillex, the Company's investigational product candidate for the treatment of Amyloid A (AA) Amyloidosis, has been selected by the Cardio-Renal Drug Product Division of the US FDA to be part of the Continuous Marketing Applications Pilot 2 programme aimed at further accelerating the development and eventual marketing of this product candidate. Under this Pilot 2 programme, each FDA division is permitted to select only one product candidate.
The selection of Fibrillex by the FDA is based in part on the fact that it has been designated as a Fast Track Product due to the life-threatening nature of AA Amyloidosis, an unmet medical need which often results in end-stage renal disease. The decision is based on other criteria as well. For example, the FDA looked at the potential value of enhanced interaction with Neurochem and emphasized the potential public health benefit from the development of the product and the likelihood that concentrated scientific dialogue could facilitate the availability of Fibrillex as a promising novel therapy. Fibrillex is presently in an on-going phase II/III clinical trial, which Neurochem expects to complete by January 2005, a release from company says.
"We are very pleased that the FDA's Cardio-Renal Division has accepted Fibrillex to be part of this new pilot project under which Neurochem and the FDA will engage in frequent scientific feedback and interaction during the development of this product candidate," said Francesco Bellini, chairman and CEO of Neurochem. "This frequent communication based on a prospectively defined agreement between the FDA and Neurochem should allow us to agree on information to be presented in our NDA submission. These on-going interactions could further expedite the FDA's review of Fibrillex," he added further.
Fibrillex has already received orphan drug status designation in the United States and Orphan Medicinal Product designation in Europe, which normally provide a drug seven and ten years of market exclusivity, respectively, upon market entry.
The Phase II/III clinical trial for Fibrillex is a two-year, international, multi-centre, randomized, double-blind, placebo-controlled, and parallel-designed trial to evaluate the safety and efficacy of Fibrillex in patients suffering from AA Amyloidosis. Neurochem has successfully completed the enrolment of 183 patients for the phase II/III trial, which is being conducted at 27 sites located across North America, Europe and Israel. Neurochem anticipates completing the trial by January 2005. To date, approximately 42 patients have completed the two-year Phase II/III trial for Fibrillex. A two-year open-label extension study for the product candidate is ongoing.
AA Amyloidosis is a progressive and fatal condition that occurs in a proportion of patients with chronic inflammatory diseases, including rheumatoid arthritis, ankylosing spondylitis, juvenile rheumatoid arthritis, and Crohn's disease.