FDA Approves a New Treatment for a Rare, Deadly Disease

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People diagnosed with Hutchinson-Gilford progeria syndrome rarely live beyond 15 years of age, and until now treatments could only target its symptoms and complications. The disease occurs when a genetic mutation changes the shape of a protein in the nuclei of a carrier’s cells. The faulty protein, called progerin, causes cells to prematurely die. Zokinvy prevents the buildup of defective progerin, thereby minimizing the damage it can do. In addition to prolonging lifespan by several years, the new drug also reduces symptoms of heart and bone problems associated with the rare condition, which affects roughly 400 children worldwide.

FDA approves first drug, Eiger’s Zokinvy, for rare rapid-aging disease

For a long time, children with the ultrarare genetic disease progeria, which causes rapid aging, didn’t have an FDA-approved therapy. But that’s about to change.

Friday, the FDA approved Zokinvy, or lonafarnib, to treat Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies in patients ages 1 and above.

Hutchinson-Gilford and progeroid laminopathies are two distinct fatal diseases marked by premature aging, with an estimated worldwide patient population of 400 and 200, respectively. Most patients die before the age of 15 from heart diseases and strokes.

Zokinvy isn’t a cure. The drug, a farnesyltransferase inhibitor, targets the toxic buildup of progerin or progerin-like farnesylated proteins.

The FDA based the approval on data from 62 patients in two single-arm clinical trials. Compared with untreated patients from a separate history study, treatment of Zokinvy increased survival by an average 2.5 years with a maximum follow-up of 11 years.

The green light gives Zokinvy developer Eiger BioPharmaceuticals a rare pediatric disease priority review voucher, which can be used to speed up FDA evaluation of a future drug. The company said it plans to sell the voucher, which, if a recent transaction from Merck is any indication, could be worth $100 million. The company will share any proceeds with the Progeria Research Foundation, which helped develop the drug.

Merck was the original developer of Zokinvy, but the company ditched its plan of spinning the drug as a cancer treatment after getting lackluster data. It has been offering the drug for free to progeroid researchers.

Eiger is also developing lonafarnib as part of a hepatitis D regimen. In the pivotal D-Liver trial, the company is pitting a combination of lonafarnib and AbbVie’s antiretroviral Norvir (ritonavir), as well as a cocktail that contains Roche’s Pegasys (pegylated interferon alpha-2a) as a third component, against placebo. Enrollment is expected to complete next year. While that trial will assess the proportion of patients who achieve at least a 2 log decline in HDV RNA and normalization of liver enzymes, another trial will evaluate sustained virologic response.

“We believe these active antivirals have a high probability of success in the trials,” BTIG analyst Bert Hazlett wrote in a Monday note.

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