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Prothena’s AL amyloidosis treatment NEOD001 gets FDA fast track status

Irish biotechnology firm Prothena has received fast track designation from the US Food and Drug Administration (FDA) for NEOD001, a new humanized monoclonal antibody for the potential treatment of amyloid light-chain (AL) amyloidosis.

NEOD001 targets the circulating soluble amyloid and deposited insoluble amyloid that accumulates in both the AL and AA forms of amyloidosis.

According to the company, this is the first investigational immunotherapy for AL amyloidosis to receive FDA fast track status.

Amyloidosis Foundation board chairman Isabelle Lousada said: "We believe this progressive disease is significantly underdiagnosed and often misdiagnosed.

"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."

The fast track status follows the company’s recent initiation of the VITAL Amyloidosis Study, a global Phase III registrational trial of NEOD001 to treat patients with AL Amyloidosis.

The international, multi-center, randomized, double-blind, placebo-controlled trial is designed to support global regulatory approvals and to enroll about 230 newly-diagnosed, treatment-naive patients with cardiac dysfunction.

During the trial, patients will be randomised on a 1:1 basis to receive 24mg/kg of NEOD001 or placebo via infusion every 28 days, with both arms receiving concurrent standard of care therapy.

Prothena chief scientific officer and head of Research and Development Gene Kinney said: "In addition to Fast Track, we have previously received orphan drug designation in both the US and EU for AL amyloidosis.

"We are delighted to have recently initiated our VITAL Phase 3 clinical trial for NEOD001 in patients with AL amyloidosis. We will continue to work closely with the FDA and European regulatory authorities to bring this therapy to patients in the most expeditious manner possible."

The company is focused on the discovery, development and commercialization of new antibodies for the potential treatment of diseases that involve amyloid or cell adhesion.