By Fineline Cube The Center for Drug Evaluation (CDE) website indicates that UK pharmaceutical major AstraZeneca’s (AZ, NASDAQ: AZN) injectable eplontersen has been tacitly approved for clinical study in hereditary and wild-type transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), aiming to reduce cardiovascular mortality and cardiovascular-related hospitalizations.
Eplontersen: An Antisense Oligonucleotide Therapy for ATTR-CM
Eplontersen, an antisense oligonucleotide (ASO) therapy developed based on the ligand conjugated antisense (LICA) technology, is co-developed by Ionis Pharmaceuticals (NASDAQ: IONS). This therapy has shown promising results in previous studies, particularly in the Phase III study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), where it met all three co-primary endpoints of improving serum transthyretin (TTR) concentration, neuropathy impairment, and quality of life, as well as all secondary endpoints. The drug is currently in a late-stage trial for ATTR-CM, further validating its potential in treating this condition.
Competition in the ATTR-CM Market
In the ATTR-CM treatment landscape, Pfizer’s Vyndamax (tafamidis) has been approved to treat ATTR-CM with National Reimbursement Drug List (NRDL) status in China. Additionally, Pfizer’s Vyndaqel (tafamidis meglumine) was approved for use in thyroxine transfering protein amyloidosis polyneuropathy, indicating a growing focus on treatments for amyloidosis-related conditions.-Fineline Info & Tech