FDA Approves Eiger Biopharma's Progeria Treatment: What Investors Should Know

The FDA approved Eiger Biopharmaceuticals Inc EIGR's lonfarnib Friday for the treatment of Hutchinson-Gilford Progeria Syndrome, commonly referred to as progeria, as well as processing-deficient progeroid laminopathies.

The Palo Alto, California-based company focuses on targeted therapies for serious rate and ultra-rare diseases. 

Lonfarnib, which will sell under the trade name Zokinvy, is a disease-modifying agent that has shown a statistically significant survival benefit in children and young adults with progeria.

Along with the approval, the FDA also issued a rare pediatric disease priority voucher to Eiger.

Separately, Eiger said it has agreed to sell the voucher for $95 million — with the company's share of the proceeds being $47.5 million — under the terms of its agreement with the Progeria Research Foundation.

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Why It's Important: Progeria and progeroid laminopathies are distinct, ultra-rare, genetic and premature aging diseases in children that often lead to mortality in young patients.

Untreated children die of heart disease at an average age of 14.5 years, Eiger said.

Zokinvy is the first FDA-approved treatment for progeria syndromes, and it is Eiger's first commercial product.

It was licensed from Merck & Co., Inc. MRK, which out-licensed the treatment without any developmental milestone payments and waived royalty obligations for a specified quantity of sales.

"The approval of this breakthrough therapy is a critical milestone for the Progeria community and also for Eiger," said Thomas Dietz, Eiger's board chairman. 

The sale of the priority review voucher provides Eiger with an important source of non-dilutive capital and further strengthens our balance sheet, the company said.

EIGR Price Action: Eiger shares were down 6.18% at $9.41 at last check Monday.

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