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Prothena Corporation
plc
, 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."
VITAL Phase 3 Registrational Trial Design
The international, multi-center, randomized, double-blind, placebo-controlled
Phase 3 study continues Prothena's commitment to provide disease-modifying
therapeutic alternatives for patients suffering from AL amyloidosis. The trial
is designed to support global regulatory approvals and to enroll approximately
230 newly-diagnosed, treatment-naïve patients with cardiac dysfunction.
Patients will be randomized on a 1:1 basis to receive 24 mg/kg of NEOD001 or
placebo via infusion every 28 days, with both arms receiving concurrent
standard of care therapy. The composite primary endpoint is event-based, with
all-cause mortality or cardiac hospitalizations as qualifying events. The trial
allows for an interim analysis to assess the primary endpoint for efficacy and
futility.
"In addition to Fast Track, we have previously received orphan drug designation
in both the U.S. and EU for AL amyloidosis. Collectively, this recognition of
NEOD001 underscores the critical need for a disease-modifying immunotherapy
specifically designed to treat patients with AL amyloidosis," said Gene Kinney,
PhD, Chief Scientific Officer and Head of Research and Development of Prothena.
"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."
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