Ionis Announces Eplontersen Receives Orphan Drug Designation from U.S. FDA for ATTR Amyloidosis

News on January 24, 2022 /BioValleyBIOON/ -- Ionis Pharmaceuticals is a leader in the field of antisense therapy. Recently, the company announced that the U.S. Food and Drug Administration (FDA) has been granted eplontersen Orphan Drug Designation (ODD):This drug is an antisense drug used to treat transthyretin (TTR)-mediated amyloidosis (ATTR).

Orphan Drug refers to a drug used for the prevention and treatment ofDiagnosisDrugs for rare diseases, which are a general term for diseases with extremely low incidence rates, also known as "orphan diseases." In the United States, rare diseases refer to disease types where the number of affected individuals is less than 200,000. Incentives for the research and development of drugs for rare diseases include various clinical development incentives, such as tax credits related to clinical trial expenses.FDAUser Fee Reduction,Clinical TrialIn DesignFDAwith the assistance, and a 7-year market exclusivity period for the approved indications after the drug's market launch.

Eplontersen is a ligand-conjugated antisense (LICA) drug that reduces the production of transthyretin (TTR) and is used to treat hereditary and non-hereditary forms.GeneticsType TTR Amyloidosis (ATTR).Currently, eplontersen is in Phase 3Clinical TrialIn tests, treat transthyretin amyloid cardiomyopathy (ATTR-CM) and transthyretin amyloid polyneuropathy (ATTR-PN). December 2021,AstraZenecaAstraZeneca collaborates with Ionis to develop and commercialize eplontersen.

ATTR Amyloidosis is a rare, systemic, progressive, and fatal disease characterized by the abnormal formation and deposition of amyloid deposits composed of misfolded transthyretin (TTR) in various organs and tissues of the human body, including peripheral nerves, heart, intestines, eyes, kidneys, central nervous system, thyroid, and bone marrow. The gradual accumulation of TTR amyloid deposits in these tissues and organs can lead to organ failure and, ultimately, death.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) and Transthyretin Amyloid Polyneuropathy (ATTR-PN) are two clinical manifestations of ATTR amyloidosis. ATTR-CM is a systemic, progressive, and fatal disease that can lead to progressive heart failure and death within four years after diagnosis. Due to a lack of disease awareness and the heterogeneity of symptoms, the disease remains under-recognized.Diagnosis, whose incidence rate is considered to be underestimated.GeneticsATTR-PN is a debilitating disease inDiagnosisIt causes peripheral nerve damage and motor dysfunction within five years, and if left untreated, death usually occurs within ten years.

Currently, several drugs have been launched, including those from Ionis Pharmaceuticals, Inc.Antisense Oligonucleotide (ASO) Drug Tegsedi(inotersen), Alnylam'sRNAi Drug Onpattro（patisiran）、PfizerTheOral transthyretin stabilizer tafamidis (Vyndamax®, Vyndaqel®)These drugs, Tegsedi and Onpattro, can target and silence specific messenger RNA (mRNA), blocking the production of TTR protein, which may help reduce deposition and promote the clearance of TTR amyloid protein in peripheral tissues, restoring the function of these tissues. Tafamidis is a first-of-its-kind TTR stabilizer that selectively binds to TTR, stabilizing the tetramer of the TTR transport protein and slowing the formation of amyloid deposits.

Among them,Pfizer's tafamidis (Vyndamax® [Vivimstat®], Vyndaqel® [Vytaltoz®]) was launched in China in 2020.In terms of regulation, another RNAi drug, vutrisiran, developed by Alnylam Pharmaceuticals is under review by the U.S. FDA. This drug is an RNAi therapy administered via subcutaneous injection and has the same mechanism of action as Onpattro. (Bioon.com)

Source of the original text:Ionis announces eplontersen receives orphan drug designation from U.S. FDA