The Amyloidosis Research Consortium, a patient led foundation, delivers draft guidance to FDA for developing drugs for AL amyloidosis.
The Amyloidosis Research Consortium (ARC) announced the submission of a draft guidance for industry on developing drugs for AL amyloidosis to the Food and Drug Administration (FDA), to improve the design of clinical trials and accelerate the review of potential therapies.
AL amyloidosis is an ultra-rare, progressive and fatal disease characterized by the accumulation of abnormal, misfolded protein (amyloid) in various tissues and organs. This amyloid protein builds up in the heart, kidneys, liver, and nervous system, resulting in multiorgan failure and death. There are no approved therapies for the treatment of patients with AL amyloidosis, or any form of systemic amyloidosis, in the United States.
Patients with AL amyloidosis are often treated off-label with drugs that are indicated for the treatment of multiple myeloma.
“We are not seeing the improvement in outcomes for patients with AL amyloidosis that we are seeing in myeloma. Therapies developed and approved specifically for AL amyloidosis are vitally needed,” said Isabelle Lousada, CEO of the Amyloidosis Research Consortium.
In September 2015, the ARC held a roundtable meeting of leading experts, industry representatives and members of the FDA to develop a pathway for drug development in amyloidosis. As a result of this roundtable meeting, it was agreed that a draft guidance document should be produced. Crafting this guidance involved active participation from all stakeholder groups including patients, medical experts, academics and biopharmaceutical industry representatives.
“Barriers to developing new medicines for rare diseases like amyloidosis stem from challenges of small study populations and an evolving understanding of disease physiology, which can make clinical trial design complex,” commented Spencer Guthrie, head of medical affairs at Prothena Biosciences. “Clear guidance from the expert community can help to optimize clinical trials to better meet the needs of patients.”
In November 2015, the ARC held a public meeting including patients, amyloidosis expert physicians from around the world, and regulators at the U.S. Food and Drug Administration (FDA). More than 240 people attended, with patients communicating the huge unmet need for new medical treatment options, the urgency to cut down lengthy development timelines and the desire to make these therapies available to patients with AL amyloidosis.
This resulted in “The Voice of the Patient” document that the ARC released earlier this year, reflecting patient’s perspectives. This publication, in concert with the draft guidance, provides multifaceted resources to improve and accelerate a drug development pathway for AL amyloidosis.
The Amyloidosis Research Consortium was established to address critical needs in clinical trials and related research for the underserved group of systemic amyloid diseases. It brings together experts in the field to address the challenges that exist in developing diagnostic tools and carrying out collaborative and innovative clinical trials. The ARC is committed to building collaborative relationships between patients, academia, industry, foundations, federal funders, and regulators to facilitate and speed new therapies to market.
The consortium is focused on increasing the amount of research in amyloidosis and building a prioritized portfolio of translational and clinical research. Its aim is to address the urgent, unmet medical needs in patients with amyloidosis. www.arci.org.
(Source: PR Newswire)