BURTON UPON TRENT, England--(BUSINESS WIRE)--
Clinigen Group plc (AIM:CLIN, ‘Clinigen’), the global pharmaceutical and services company, has partnered with Eiger BioPharmaceuticals, Inc. (NASDAQ:EIGR, ‘Eiger’) to launch a worldwide lonafarnib Managed Access Program for patients with Progeria and Progeroid Laminopathies.
Progeria, also known as Hutchinson-Gilford Progeria Syndrome, is a rare and fatal genetic condition of accelerated aging in children that researchers believe is caused by a genetic mutation that results in an overabundance of the farnesylated aberrant protein named progerin. Signs of Progeria are typically observed within the first two years of life and include growth failure, loss of body fat and hair, aged-looking skin, stiffness of joints, hip dislocation, generalised atherosclerosis, cardiovascular (heart) disease and stroke.
Lonafarnib is a first-in-class, orally active inhibitor of farnesyltransferase, an enzyme involved in the modification of proteins through a process called prenylation. Lonafarnib has been granted Orphan Drug designation for Progeria by FDA and EMA and Breakthrough and Rare Pediatric Disease designation by FDA. Eiger BioPharmaceuticals is currently preparing an NDA with plans to file in 2019.
Healthcare professionals can obtain details about the lonafarnib Managed Access Program by calling the customer service team at 824 123 (RoW) or 0 (UK) or emailing ; or calling The Progeria Research Foundation at 978-535-2594 or emailing .
Notes to Editors
About Progeria
Progeria, also known as Hutchinson-Gilford
Progeria Syndrome (HGPS), is a rare and fatal genetic condition of
accelerated aging in children. Progeria is caused by a point mutation in
the LMNA gene, encoding the lamin A protein, yielding the
farnesylated aberrant protein, progerin. Lamin A protein is part of the
structural scaffolding that holds the nucleus together. Researchers now
believe that progerin may make the nucleus unstable, and that cellular
instability may lead to the process of premature aging in Progeria.
Children with Progeria die of the same heart disease that affects
millions of normally aging adults (arteriosclerosis), but at 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. It is estimated
that 400 children worldwide have Progeria.
About Progeroid Laminopathies
Progeroid Laminopathies are
genetic conditions of accelerated aging caused by a constellation of
mutations in the lamin A and/or Zmpste24 genes yielding farnesylated
proteins that are distinct from progerin. While non-progerin producing,
these genetic mutations result in disease manifestations with phenotypes
that have overlap with, but are distinct, from Progeria. Collectively,
worldwide prevalence of Progeroid Laminopathies is likely greater than
Progeria.
About Lonafarnib
Lonafarnib is a well-characterized,
late-stage, orally active inhibitor of farnesyltransferase, an enzyme
involved in modification of proteins through a process called
prenylation. Progerin is a farnesylated protein that researchers believe
cannot be cleaved, resulting in tight association with the nuclear
envelope, which is believed to lead to changes in nuclear envelope
morphology and subsequent cellular damage. Lonafarnib blocks the
farnesylation of progerin and has been dosed in over 80 children with
Progeria at Boston Children’s Hospital in Phase 1/2 and Phase 2 studies
funded by The Progeria Research Foundation ().
Lonafarnib has been granted Orphan Drug designation for Progeria by the FDA and EMA and Breakthrough and Rare Pediatric Disease designation by the FDA. Lonafarnib is not approved for any indication, and is licensed by Eiger from Merck Sharp & Dohme Corp.
About Clinigen Group
Clinigen Group plc (AIM:CLIN) is a
global pharmaceutical and services company with a unique combination of
businesses focused on providing ethical access to medicines. Its mission
is to deliver the right medicine to the right patient at the right time
through three areas of global medicine supply; clinical trial,
unlicensed and licensed medicines. The Group has sites in North America,
Europe, Africa and Asia Pacific. In October 2018, the Group acquired
CSM, a specialist provider of packaging, labelling, warehousing and
distribution services, with sites in the US and Europe, and iQone, a
specialist pharmaceutical company in Switzerland.
For more information, please visit .
About Eiger BioPharmaceuticals, Inc.
Eiger is a late-stage
biopharmaceutical company focused on the accelerated development and
commercialization of a pipeline of targeted, first-in-class therapies
for rare and ultra-rare diseases. The company’s lead program is in Phase
3, developing lonafarnib, a first-in-class prenylation inhibitor for the
treatment of Hepatitis Delta Virus (HDV) infection. The company is also
preparing an NDA with plans to file in 2019 for lonafarnib in the
treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and
Progeroid Laminopathies. For additional information about Eiger, please
visit .
About The Progeria Research Foundation
The Progeria Research
Foundation (PRF) was established in 1999 by Leslie Gordon, MD, PhD and
Scott Berns, MD, MPH, parents of a child with Progeria. In 2003 the PRF
Genetics Consortium, led by Francis Collins, MD, PhD, isolated the
Progeria gene. PRF has been the driving force behind studies to evaluate
lonafarnib as a potential treatment for Progeria. PRF has developed
programs and services to aid those affected by Progeria and the
scientists who conduct Progeria research. Today, PRF is the only
non-profit organization solely dedicated to finding treatments and a
cure for Progeria. For more information, please go to .
View source version on businesswire.com:
