Eiger BioPharmaceuticals Announces FDA Acceptance of NDA for Filing with Priority Review of Zokinvy™ (lonafarnib) for Treatment of Progeria and Progeroid Laminopathies
"The acceptance of our first NDA is a significant milestone for Eiger, and an important step toward bringing a treatment to children and young adults with Progeria and Progeroid Laminopathies," said
The Zokinvy NDA includes data from a study published by Gordon et al in
"This milestone is the culmination of twelve years of clinical trials, treating children from over 30 countries and six continents with Progeria and Progeroid Laminopathies," stated
Progeria, also known as Hutchinson‐Gilford Progeria Syndrome (HGPS), is an ultra-rare and fatal genetic condition of accelerated aging in children. Progeria is caused by a point mutation in the LMNA gene, which encodes the lamin A protein, yielding the farnesylated aberrant protein, progerin. Lamin A protein is part of the structural scaffolding that holds the nucleus together. Without lonafarnib therapy, 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 similar to Progeria.
About Zokinvy (lonafarnib)
Zokinvy 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, aberrant protein that researchers believe cannot be cleaved, resulting in tight association with the nuclear envelope, which leads to changes in nuclear envelope morphology and subsequent cellular damage.
Zokinvy blocks the farnesylation of progerin and has been dosed in over 90 children with Progeria at
Zokinvy has been granted Orphan Drug Designation for Progeria by the FDA and EMA and Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the FDA. Zokinvy (lonafarnib) is not approved for any indication, and is licensed by Eiger from
FDA has conditionally accepted the proposed brand name Zokinvy™ for lonafarnib for treatment of Progeria and Progeroid Laminopathies. Final approval of the Zokinvy brand name is conditional on FDA approval of the product candidate, lonafarnib.
Eiger is a late-stage biopharmaceutical company focused on the development and commercialization of first-in-class, well-characterized drugs for serious rare and ultra-rare diseases for patients with high unmet medical needs, for which no approved therapies exist.
The NDA and MAA submissions for Zokinvy for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) and Progeroid Laminopathies have been accepted for filing. Eiger has also established a global Managed Access Program, expected to span greater than 40 countries, to ensure all children and young adults with Progeria and Progeroid Laminopathies have access to treatment.
The company's lead program is in Phase 3, developing lonafarnib, a first-in-class oral prenylation inhibitor for the treatment of Hepatitis Delta Virus (HDV) infection. The company is also advancing peginterferon lambda, a first-in-class interferon, toward registration for the treatment of HDV. For additional information about Eiger and its clinical programs, please visit www.eigerbio.com.
Note Regarding Forward-Looking Statements
This press release contains "forward-looking" statements that involve substantial risks and uncertainties. All statements other than statements of historical facts, including statements regarding our future financial condition, timing for and outcomes of clinical results, business strategy and plans and objectives for future operations, are forward-looking statements. These forward-looking statements include terminology such as "believe," "will," "may," "estimate," "continue," "anticipate," "contemplate," "intend," "target," "project," "should," "plan," "expect," "predict," "could," "potentially" or the negative of these terms. 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, including the potential for approval of our Zokinvy product candidate in the
These statements concern product candidates that have not yet been approved for marketing by the
Various important factors could cause actual results or events to differ materially from the forward-looking statements that Eiger makes, including the risks described in the "Risk Factors" sections in the Quarterly Report on Form 10-Q for the quarter ended
Investors and Media:
Sr VP, Clinical Development
View original content to download multimedia:http://www.prnewswire.com/news-releases/eiger-biopharmaceuticals-announces-fda-acceptance-of-nda-for-filing-with-priority-review-of-zokinvy-lonafarnib-for-treatment-of-progeria-and-progeroid-laminopathies-301062068.html