Eiger nabs the first FDA approval for Progeria, an ultra-rare premature aging disease, with an old Merck drug
Eiger BioPharmaceuticals $EIGR has received an FDA OK for a drug Merck licensed to them at no cost — and now reportedly plans to charge a level consistent with other ultra-rare disease medicines.
The biotech announced Friday evening that regulators had approved lonafarnib for the treatment of Hutchinson-Gilford progeria syndrome, also known as Progeria, as well as some progeroid laminopathies in children older than one year. It’s the first approval granted for the condition, and the drug will be marketed and sold as Zokinvy.
Investors met the news warmly, sending the stock up roughly 12% in post-market trading Friday. By early Monday morning, the price had leveled off to up about 5%.
One of the rarest diseases in the world with an estimated 350 to 400 cases at any one time, Progeria is a genetic disorder that causes premature aging in children. About 180 individuals with the disease have been identified, Eiger says, including 20 in the US and 23 in Europe. Those diagnosed with the disease have an average life expectancy of about 14 years and generally suffer from complications such as heart failure, stroke or hardening of the arteries.
Lonafarnib, an oral farnesyltransferase inhibitor, helps prevent the buildup of defective progerin or progerin-like proteins. Two clinical trials enrolling 62 patients have shown a reduction in mortality by 60 percent (p=0.0064), and children taking the drug have lived about 2.5 years longer on average after the 11-year follow-up period.
With the approval, Eiger also won a Priority Review Voucher for rare pediatric diseases, which it plans to sell. The biotech will split the proceeds 50-50 with the Progeria Research Foundation, with whom it developed the drug.
Eiger licensed the drug in 2010 for anti-viral indications, including Hepatitis D, after Merck shut down an investigative program for cancer. The companies expanded their collaboration in 2018 to include Progeria, giving Eiger full commercial and distribution rights for any licensed and approved indications. Eiger was responsible for all regulatory, commercialization and distribution aspects of lonafarnib, and Merck will not receive any milestone payments on Progeria sales.
The biotech now plans to price the drug at levels comparable to other ultra-rare disease medicines, according to the Boston Globe, and does not expect “cost to be a barrier to patient access.” Eiger did not respond to an Endpoints News request for comment on pricing early Monday morning.
Lonafarnib’s approval proves a big win for Eiger following a rough couple of years in which two trials for ubenimex flopped in pulmonary arterial hypertension and lower leg lymphedema. Eiger has scrapped work for that medicine unless another company expresses interest in developing it. In 2018, Eiger saw some good news when its avexitide program returned positive Phase II results in post-bariatric hypoglycemia, or low blood glucose levels experienced after weight-loss surgery.
The company is also conducting a Phase III trial for lonafarnib in Hepatitis D, as well as a Phase III for peginterferon lambda in the same indication.