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First Investigational Immunotherapy for AL Amyloidosis to Receive FDA Fast Track Designation – Follows Recent Initiation of The VITAL Amyloidosis Study, a Global Phase 3 Registrational Trial of NEOD001 for Patients with AL Amyloidosis
Prothena Corporation plc (Nasdaq:PRTA), a late-stage clinical biotechnology company focused on the discovery, development and commercialization of novel antibodies for the potential treatment of diseases that involve amyloid or cell adhesion, today announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to NEOD001, a novel monoclonal antibody for the potential treatment of AL amyloidosis.
This is the first investigational immunotherapy specifically targeting the disease-causing protein in AL amyloidosis to receive Fast Track designation.
The FDA’s Fast Track Drug Development Program is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. An unmet medical need is a condition whose treatment or diagnosis is not addressed adequately by available therapy. The purpose of the Fast Track designation is to make important new drugs available to patients earlier.
The Fast Track program also provides a company with the ability to submit sections of the Biologics License Applications (BLA) for review before the company submits the complete BLA. This enables the FDA to review sections of the BLA as they are received, rather than waiting until every section of the application is completed, and also allows for Priority Review, shortening the standard review of the final BLA to six months. A drug program with Fast Track designation permits the company to have early and frequent communications with the FDA in the development and review of the product candidate, potentially leading to faster drug approval.
“The amyloidosis community has long hoped for a drug or treatment that would remove the amyloid deposits from the involved organs,” said Muriel Finkel, President of the Amyloidosis Support Groups Inc. “Prothena’s published results, and those recently presented, as well as excitement generated from amyloidosis patients, provide us hope that NEOD001 may offer us this very solution for our AL amyloidosis patients.”
“We believe this progressive disease is significantly underdiagnosed and often misdiagnosed,” commented Isabelle Lousada, Board Chairman of the Amyloidosis Foundation. “Until now, there have been no therapeutics developed specifically to treat patients with AL amyloidosis. Treatment has been limited to unapproved use of chemotherapeutic agents, which do not address the underlying cause of the disease and may have significant side effects. We welcome Prothena’s dedication to pursuing a safe and effective therapy, and are encouraged by the safety profile of NEOD001 and the organ response rate results seen to date.”
