Last Updated on December 31, 2023 by The Health Master
Carlsbad: Ionis Pharmaceuticals, Inc. has recently announced that the U.S. Food and Drug Administration (USFDA) has granted orphan drug designation to eplontersen, an investigational antisense medicine for the treatment of people living with transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition.
An orphan drug designation is granted by the USFDA to drugs and biologics intended for treatment, prevention, or diagnosis of a rare disease or condition that affects fewer than 200,000 people in the U.S. at the time of designation.
Eplontersen is a LIgand-Conjugated Antisense (LICA) investigational medicine currently in Phase 3 clinical trials for amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN).
It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).
In December 2021, Ionis announced a strategic collaboration with AstraZeneca to develop and commercialize eplontersen.
ATTR-CM is a systemic, progressive, and fatal condition that leads to progressive heart failure and death within four years of diagnosis. It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.
Without treatment, ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.
Under the USFDA’s Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants, and waivers of certain administrative fees for clinical trials, as well as seven years of market exclusivity following drug approval.
“Receiving USFDA orphan drug status for eplontersen underscores the significant unmet need for novel treatment options for people living with transthyretin-mediated amyloidosis.
We look forward to working closely with regulators, clinical investigators, patients and their families to advance this important medicine and make it available to those who may benefit from it,” said Richard S. Geary, Ph.D., executive vise president and chief development officer at Ionis.
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