- First and only treatment approved in Europe to treat Hutchinson-Gilford progeria
syndrome and processing-deficient progeroid
laminopathies1
- Extended life by an average of 4.3 years in children and
young adults with HGPS2
- Now approved for use in 30 European countries, following
approval in the U.S in 20201
PALO
ALTO, Calif., July 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 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.
About progeria
Collectively known as progeria, Hutchinson-Gilford progeria
syndrome and progeroid laminopathies are ultra-rare, fatal, genetic
premature aging diseases that accelerate mortality in young
patients. There are approximately 20 children and young adults with
progeria identified across Europe.
HGPS is caused by a point mutation in the LMNA gene, yielding
the farnesylated aberrant protein, progerin. Progeroid
laminopathies are genetic conditions of accelerated aging caused by
a constellation of mutations in the LMNA and/or ZMPSTE24 genes
yielding farnesylated proteins that are distinct from
progerin.4,5
Without Zokinvy therapy, children with HGPS commonly die of the
same heart disease that affects millions of normally aging adults
(arteriosclerosis), by an average age of 14.5 years. Disease
manifestations include severe failure to thrive, scleroderma–like
skin, global lipodystrophy, alopecia, joint contractures, skeletal
dysplasia, global accelerated atherosclerosis with cardiovascular
decline, and debilitating strokes.3
About Zokinvy®
(lonafarnib)
Zokinvy is a first-in-class disease-modifying agent that
blocks the accumulation of defective progerin and progerin-like
proteins which leads to cellular instability and premature aging in
children and young adults with progeria. Zokinvy has demonstrated a
statistically significant survival benefit in children and young
adults with HGPS.1,4
The most commonly reported adverse reactions were
gastrointestinal (vomiting, diarrhea, nausea), and most were mild
or moderate (Grade 1 or 2) in severity. Many progeria patients have
received continuous Zokinvy therapy for more than 10
years.1,2
Eiger licensed exclusive worldwide rights to Zokinvy from
Merck, known as MSD, outside of the
United States and Canada.
Merck will not receive any milestone payments for the development
of Zokinvy for the treatment of progeria and has waived royalty
obligations from Eiger for a specified quantity of Zokinvy.
Zokinvy EU indication
In Europe, Zokinvy is indicated
for the treatment of patients 12 months of age and older with a
genetically confirmed diagnosis of Hutchinson-Gilford progeria
syndrome or a processing-deficient progeroid laminopathy associated
with either a heterozygous LMNA mutation with progerin-like protein
accumulation or a homozygous or compound heterozygous ZMPSTE24
mutation.
For the labeled safety information for Zokinvy please see the
full European Summary of Product Characteristics here.
For Important Safety Information and prescribing information for
Zokinvy in the U.S., please visit www.zokinvy.com.
About Eiger
Eiger is a commercial-stage biopharmaceutical company focused on
the development of innovative therapies to treat and cure hepatitis
delta virus (HDV) and other serious diseases. The Eiger HDV
platform includes two first-in-class therapies in Phase 3 that
target critical host processes involved in viral replication. Eiger
is also developing peginterferon lambda as a therapeutic for
COVID-19 and reported positive results from TOGETHER, a
Phase 3 investigator-initiated study.
All five Eiger rare disease programs have been granted FDA
Breakthrough Therapy designation: lonafarnib and peginterferon
lambda for HDV, Zokinvy for progeria, and avexitide for both
congenital hyperinsulinism and post-bariatric hypoglycemia.
For additional information about Eiger and its clinical
programs, please visit www.eigerbio.com.
About Progeria Family
Circle
The Progeria Family Circle (PFC) is a European patient network,
founded in 1997, supporting children and young adults with progeria
and their families on a European level. The PFC goal is to support
better and faster recognition of symptoms of progeria, raise
awareness for a better acceptance of progeria children and young
adults in public communities, provide opportunities for families to
meet each other through many annual reunions throughout
Europe, and offer other necessary
and permanent support for families with children with
progeria.
For more information, please visit
www.progeriafamilycircle.blogspot.com.
Forward-looking
statements
This press release contains forward-looking statements within
the meaning of the safe harbor provisions of the U.S. Private
Securities Litigation Reform Act of 1995. All statements other than
statements of historical facts, including statements regarding our
future financial condition, timing for and outcomes of clinical
results, prospective products, preclinical and clinical pipelines,
regulatory objectives, business strategy and plans and objectives
for future operations, are forward looking statements.
Forward-looking statements are our current statements regarding our
intentions, beliefs, projections, outlook, analyses or current
expectations concerning, among other things, the timing of our
ongoing and planned clinical development across our pipeline and
the potential for success of any of our products or product
candidates. Various important factors could cause actual results or
events to differ materially from the forward-looking statements
that Eiger makes, including additional applicable risks and
uncertainties described in the "Risk Factors" sections in the
Annual Report on Form 10-Q for the quarter ended March 31, 2022 and Eiger's subsequent filings
with the SEC. The forward-looking statements contained in this
press release are based on information currently available to Eiger
and speak only as of the date on which they are made. Eiger does
not undertake and specifically disclaims any obligation to update
any forward-looking statements, whether as a result of any new
information, future events, changed circumstances or otherwise.
CONTACTS:
Investors:
Sylvia
Wheeler
Wheelhouse Life Science Advisors
swheeler@wheelhouselsa.com
Media:
Sarah
Mathieson
SVP, Corporate Affairs
smathieson@eigerbio.com
References:
- Data on file, Eiger BioPharmaceuticals.
- Summary of Product Characteristics, July 2022.
- Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford
Progeria Syndrome. 2003 Dec 12 [Updated 2019 Jan 17]. In: Adam MP,
Ardinger HH, Pagon RA, et al., editors. GeneReviews®
[Internet]. Seattle (WA):
University of Washington, Seattle;
1993-2020.
- Gordon LB, Shappell H, Massaro J, et al. Association of
lonafarnib treatment vs no treatment with mortality rate in
patients with Hutchinson-Gilford progeria syndrome. JAMA.
2018;319(16):1687-1695. doi:10.1001/jama.2018.3264.
- Marcelot A, Worman HJ, and Zinn-Justin S. Protein structural and
mechanistic basis of progeroid laminopathies. FEBS Journal.
2021:288:2757-2772. Doi:10.111/febs.15526.
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SOURCE Eiger BioPharmaceuticals, Inc.