Eiger Receives Positive CHMP Opinion for Zokinvy as a Treatment for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies – QNT Press Release


  • If authorized, Zokinvy® (lonafarnib) will be the first and only treatment approved in Europe to treat Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies – collectively known as progeria.
  • CHMP based its opinion on the results of data demonstrating Zokinvy increased survival by 4.3 years in children and young adults with HGPS.
  • Zokinvy was approved by the US FDA as the first and only treatment for progeria in November 2020.

PALO ALTO, Calif., May 20, 2022 /PRNewswire/ — Eiger BioPharmaceuticals Inc. (NASDAQ:EIGR), a commercial-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure hepatitis delta virus and other serious diseases, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the European Commission approve Zokinvy, a first-in-class breakthrough therapy for Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).

HGPS and PL are devastating, ultra-rare, and fatal pediatric diseases that cause dramatically accelerated aging and premature death. The main cause of death from these conditions is heart attack or stroke due to severe hardening of the arteries. Without Zokinvy treatment, children with HGPS die at an average age of 14.5 years.

The CHMP based its decision on the results of two clinical trials which showed that Zokinvy, a disease-modifying agent, lowered the risk of death in children with HGPS by 72%. Zokinvy extended life by an average of 4.3 years in children and young adults with HGPS. The science and innovation for Zokinvy spans 13 years of clinical research, including clinical trials involving almost 100 children diagnosed with progeria from 37 different countries across six continents. The US approval and positive CHMP opinion are the result of a pioneering partnership between Eiger and the Progeria Research Foundation to bring Zokinvy to market and hope to patients.

“If authorised, Zokinvy will represent the only therapeutic option that has been proven to meaningfully extend the lives of children with HGPS – with the significant effect of extending their average life span by nearly one third,” said Prof. Thorsten MarquardtPaediatric and Adolescent Medicine, University of Münster. “The development of therapies for ultra-rare diseases like progeria is often ignored leaving patients, families, and physicians to cope alone, so progress like this is extremely impactful to the entire rare disease community. “

“Today’s positive opinion from the CHMP brings us one step closer to making Zokinvy available to help HGPS patients in Europe live longer lives. With this news, our recently announced partnership agreement in Japanand our ongoing commercialization in the US, we are executing our strategy to give hope to vulnerable patients around the world,” said David CoryPresident and CEO, Eiger. “Additionally, Eiger continues to drive positive momentum by advancing our late-stage pipeline of breakthrough therapies which include much needed potential treatments for devastating global diseases like hepatitis delta virus, congenital hyperinsulinism, and COVID-19.”

Based on the CHMP recommendation, a decision by the European Commission is anticipated within approximately two months. If granted, the centralized marketing authorization would be valid in all 27 EU member states as well as the United Kingdom, Iceland, Liechtensteinand Norway. Zokinvy is expected to be made available commercially following successful completion of reimbursement discussions country-by-country.

“On behalf of the progeria community of children, families, and healthcare providers across EuropeI am extremely pleased with the CHMP’s decision today. This important milestone brings us closer to the first approved treatment for progeria in Europe,” added Maryet Stamsnijder, President, Progeria Family Circle. “Importantly, it also helps us build much-needed awareness of progeria to better support families impacted by this devastating disease as well …

Full story available on Benzinga.com



Source link

Recommended For You

About the Author: News Center