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Fibrillex Selected for New FDA Pilot 2 Program
The selection of Fibrillex(TM) 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(TM) as a promising novel therapy. Fibrillex(TM) is presently in an on-going Phase II/III clinical trial which Neurochem expects to complete by January 2005.
"We are very pleased that the FDA's Cardio-Renal Division has accepted Fibrillex(TM) 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, Ph. D., 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(TM)."
Fibrillex(TM) 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(TM) is a two-year, international, multi-center, randomized, double-blind, placebo-controlled, and parallel-designed trial to evaluate the safety and efficacy of Fibrillex(TM) in patients suffering from AA Amyloidosis. Neurochem has successfully completed the enrollment 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(TM). A two-year open-label extension study for the product candidate is on-going.
Fibrillex(TM) is an oral product candidate for the treatment of AA Amyloidosis through the prevention of amyloid fibril formation. Fibrillex(TM) belongs to the class of glycosaminoglycan (GAG) mimetics, better known as disease-modifiers. By mimicking GAGs, Fibrillex(TM) competitively binds to the AA protein, preventing natural GAGs from binding to the same AA protein. Fibrillex(TM) is expected to prevent AA fibril formation and the deposition of AA fibrils in organs.
About AA Amyloidosis
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. The disease also occurs in patients suffering from many other conditions ranging from chronic infections to inherited inflammatory diseases such as Familial Mediterranean Fever. The most common clinical presentation of AA Amyloidosis is kidney malfunction. Involvement of the gastrointestinal system is also frequent and is usually manifested as chronic diarrhea, gastrointestinal bleeding, abdominal pain and malabsorption. Enlargement of the liver and the spleen may also occur in some patients.
This disease has a poor prognosis, with a five-year survival rate of approximately 50%.(1) Left untreated, patients gradually progress to end-stage renal failure, which is the cause of death in at least 35% of the cases.(2, 3)
(1) Pepys, M., et al. Amyloidosis. Oxford Textbook of Medicine (4th ed.) Oxford: Oxford University Press 2003; 162-73.
(2) Gertz, M.A., et al. Medicine (Baltimore) 1991 Jul: 70(4); 246-56.
(3) Joss, N., et al. QJMed. 2000: 93; 535-42.