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 the European Commission (EC) has granted marketing authorization for Zokinvy® (lonafarnib), a first-in-class breakthrough therapy for children and young adults with Hutchinson-Gilford progeria syndrome (HGPS) and processing-deficient progeroid laminopathies (PL).
Collectively known as progeria, HGPS and PL
are devastating ultra-rare, and fatal pediatric diseases that cause
dramatically accelerated aging and premature death. The main cause of death 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.3,4
“Until now, no medication has been approved
in Europe for people living with this debilitating condition. For the first
time we have a therapeutic option with Zokinvy which has been proven to
meaningfully extend the average life span of children with HGPS by nearly one
third,” said Professor Thorsten Marquardt, Paediatric and Adolescent Medicine,
University of Münster, Germany. “With less than 5% of rare diseases having an
approved treatment, the authorization of Zokinvy represents both a significant
medical milestone and increased hope for the progeria community.”
The EC authorization follows the positive
opinion granted by European Committee for Medicinal Products for Human Use
(CHMP) in May 2022 which was based on the positive results of two pivotal
clinical trials demonstrating that Zokinvy, an oral disease-modifying agent
which targets the cause of progeria, lowered 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.2
“The European Commission’s approval of
Zokinvy, Eiger’s first approval in Europe, is the culmination of years of
dedication and commitment by the Eiger team and the Progeria Research
Foundation, our partners in this pioneering endeavor,” said David Cory,
President and CEO, Eiger. “Eiger’s mission is to bring innovative medicines to
underserved patients around the world. We are proud of this approval which
further demonstrates our ability to partner with investigators and regulators
to achieve that mission.”
“The approval of Zokinvy is extremely welcome
news for families and the entire progeria community,” added Marjet Stamsnijder, President, Progeria
Family Circle. “I am delighted we now have a treatment approved for use in
Europe that has the ability to transform the lives of children and young adults
with progeria.”
The European Commission’s centralized
marketing authorization is valid in all 27 EU member states as well as Iceland,
Liechtenstein, and Norway. Regulatory review is ongoing by the UK’s Medicine and Healthcare
products Regulatory Agency (MHRA) as part of the European Commission
Decision Reliance Procedure. Eiger expects to make Zokinvy
available following successful completion of reimbursement discussions
country-by-country.