Pharma Focus Asia

Eiger and Partner, AnGes, Gain Japan Approval for Zokinvy® in Progeria Syndromes

Friday, January 19, 2024

Eiger BioPharmaceuticals, Inc. (Nasdaq: EIGR) has recently announced that it, in collaboration with AnGes, Inc., has secured marketing approval from the Ministry of Health, Labour and Welfare in Japan for Zokinvy (lonafarnib). Zokinvy is a treatment developed for Hutchinson-Gilford progeria syndrome (HGPS) and processing deficient progeroid laminopathy (PDPL), rare metabolic diseases.

David Apelian, MD, PhD, MBA, CEO of Eiger, expressed satisfaction with the approval, stating, "We and our partner, AnGes, are pleased that Zokinvy is now approved in Japan for patients living with progeria, an ultra-rare and fatal pediatric disease that can result in premature death. We would like to thank the Progeria Research Foundation for their continued support of the regulatory submission as well as the patients and their families."

Progeria, encompassing HGPS and PDPL, are extremely rare and fatal pediatric diseases that cause accelerated aging and premature death. The primary cause of death in affected individuals is heart attack or stroke due to severe hardening of the arteries.

The approval in Japan was based on positive results from two pivotal clinical trials, demonstrating that Zokinvy, an oral disease-modifying agent addressing the root cause of progeria, reduced the risk of death in children by 72% and extended life by an average of 4.3 years in children and young adults with HGPS.

Hutchinson-Gilford progeria syndrome is caused by a point mutation in the LMNA gene, leading to the production of the farnesylated aberrant protein, progerin. Progeroid laminopathies result from mutations in the LMNA and/or ZMPSTE24 genes, yielding farnesylated proteins distinct from progerin.

Zokinvy is a first-in-class disease-modifying agent designed to inhibit the accumulation of defective progerin and progerin-like proteins, preventing cellular instability and premature aging in affected individuals. The drug has shown a statistically significant survival benefit in children and young adults with HGPS.

Commonly reported adverse reactions to Zokinvy include mild to moderate gastrointestinal issues such as vomiting, diarrhea, and nausea. Many progeria patients have received continuous Zokinvy therapy for more than 10 years.

Zokinvy is FDA approved for the treatment of patients aged 12 months and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with specific mutations.

Eiger and AnGes entered into an exclusive distribution agreement for the treatment of HGPS and PDPL indications, Zokinvy (Lonafarnib), in Japan on May 10, 2022. In March 2023, the Ministry of Health, Labour and Welfare designated Zokinvy as an orphan drug. 



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