FDA Approves a New Treatment for a Rare, Deadly Disease

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Eiger’s progeria drug nabs speedy review

Children with progeria age too quickly, developing symptoms common in much older people such as heart disease and joint stiffness. Treatments focus on managing symptoms, but the FDA could approve a drug that changes the course of the rare, genetic disease later this year.

Eiger BioPharmaceuticals scored a priority review tag from the agency for its progeria prospect, lonafarnib, with a target action date of Nov. 22. Merck originally developed the drug for cancer but ditched that plan after it turned up lackluster results. It has provided lonafarnib to progeria researchers, including Eiger, for free since the early 2010s. Eiger picked up the commercialization and distribution rights to lonafarnib in 2018.

“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 Eiger President and CEO David Cory in a statement. The company is gearing up to launch lonafarnib under the name Zokinvy in the U.S. and Europe.

Progeria is caused by mutations in the LMNA gene, which codes for a protein that helps determine the shape of the nucleus within cells. Children with the mutation produce a produce an abnormal version of that protein called progerin, which makes the membrane around a cell’s nucleus unstable and leads to the cell’s premature death. Progeria has many symptoms, from skeletal defects and alopecia to loss of the fat layer under the skin, but most patients will die from heart disease in their teens.

Lonafarnib works by blocking an enzyme that modifies progerin. It has been tested in more than 90 children in phase 1 and 2 studies. The New Drug Application includes data from a study that compared results from 27 patients who took lonafarnib to a group of 103 patients who did not. After two years, the study found that 3.7% of the patients who took lonafarnib died compared to 33.3% of the untreated patients.

Eiger is seeking approval for lonafarnib in progeroid laminopathies as well as progeria. Like progeria, the former are a group of genetic conditions that cause accelerated aging, but they are caused by modified proteins other than progerin.


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