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Last week, families of children and young adults with Progeria welcomed the news that we are one step closer to the first approved treatment for this ultra-rare, devastating condition that causes premature aging in children. Children with Progeria, also known as Hutchinson‐Gilford Progeria Syndrome (HGPS), die of the same heart disease that affects millions of normally aging adults (arteriosclerosis), but at an average age of 14 years.

Progeria is caused by a spontaneous mutation in a gene that encodes a protein called lamin A that is critical for the structural scaffolding that holds the nuclei of cells together. Children with Progeria produce an abnormal lamin A protein called progerin, which accumulates in the nucleus of cells, causing a failure to thrive, scleroderma‐like skin, global lipodystrophy, joint contractures, accelerated arteriosclerosis with cardiovascular decline, and debilitating strokes, which are the most common cause of mortality. Progeroid Laminopathies are a related, but heterogeneous set of genetic conditions with disease manifestations that can overlap with Progeria.

Prevalence of Progeria is 1 in 20 million. Worldwide, it is estimated that there are over 400 children and young adults with Progeria and Progeria Laminopathies. Currently, over 160 patients have been identified in over 40 countries around the world.

There is currently no approved treatment for Progeria and Progeroid Laminopathies.

Lonafarnib: A potential treatment for Progeria and Progeroid Laminopathies

Lonafarnib blocks the production of progerin and progerin-like proteins and has been dosed in over 90 children and young adults with Progeria and Progeroid Laminopathies across multiple clinical studies. In a study published in the Journal of the American Medical Association (JAMA) in 2018, a survival analysis demonstrated that treatment with lonafarnib alone compared with no treatment was associated with a significantly lower mortality rate (3.7% vs. 33.3%) and a reduction in the risk of mortality of 88% after an average of 2.2 years of follow up. Lonafarnib was generally well tolerated. Most commonly reported side effects were gastrointestinal in nature.

Eiger BioPharmaceuticals and Lonafarnib

Eiger (NASD: EIGR) is a late-stage biopharmaceutical company focused on the development and commercialization of treatments for serious rare and ultra-rare diseases, located in Palo Alto, California. Eiger’s lead program is the development of lonafarnib in Hepatitis Delta Virus (HDV) infection, the deadliest form of human viral hepatitis. Eiger is currently enrolling and dosing HDV-infected patients with lonafarnib in D-LIVR, a global Phase 3 study to support approval.

When Eiger CEO David Cory became aware that there was another rare disease that had shown benefit with lonafarnib treatment, the company made a commitment to provide drug for Progeria patients in clinical trials, and to identify a path to gain regulatory approval such that a treatment would be available for children and young adults with Progeria. “Eiger is the only manufacturer of lonafarnib. Ensuring that patients with Progeria have access to the only drug that has demonstrated a survival benefit is the right thing to do,” stated David Cory.

To this end, Eiger recently submitted a New Drug Application (NDA) to the Food and Drug Administration (FDA) and a Market Authorization Application (MAA) to the European Medicines Agency (EMA) for lonafarnib for the treatment for Progeria and Progeroid Laminopathies. Approvals by FDA and EMA are expected as soon as late 2020. In addition to seeking approval in the U.S. and Europe, Eiger has established a global Managed Access Program to make lonafarnib available to children and young adults in over 40 countries where lonafarnib has not yet been or may never be approved. Many patients with Progeria or Progeroid Laminopathies have received continuous lonafarnib treatment for greater than 10 years in clinical trials and through Eiger’s Managed Access Program.

“We are committed to the Progeria community, to bring the first approved treatment to children and young adults with Progeria and Progeroid Laminopathies,” said David Cory, President and CEO of Eiger.  “The submission of both the MAA and NDA are important steps to achieving this goal, and the global Managed Access Program, funded and managed by Eiger, ensures availability of lonafarnib to patients living in many countries beyond the U.S. and Europe. We would like to thank all the children and young adults with Progeria and their families for their persistence and perseverance.”

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