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Pharming Group NV, a Dutch biotech company that uses the milk of so-called "magic rabbits" to make its lead product Ruconest, intends to broaden the medicine's use beyond a rare genetic disease to address a market worth some $5.6 billion, piquing the interest of Big Pharma, according to analysts.
Ruconest was approved five years ago by U.S. regulators to treat hereditary angioedema, or HAE, a rare protein deficiency disorder that affects about 7,000 patients in the U.S. and is characterized by debilitating attacks of swelling in the organs, airways, hands or feet.
Administered via intravenous injection for acute episodes, Ruconest contains a recombinant protein that is produced in the milk of transgenic rabbits. These lactating animals are housed in a controlled environment in the Netherlands where they are used as a type of bioreactor, said CEO Sijmen de Vries. One cup of milk per day extracted from these magic rabbits provides enough protein to make one and a half doses of Ruconest. The Leiden, Netherlands-based company now intends to extract the protein from the milk of cows, given that 350 rabbits can produce the same amount as one cow, as it seeks to expand into other indications.
Having turned a profit for the first time ever in 2018 thanks to rising sales of Ruconest in the U.S. and in Europe, Pharming is also exploring new ways of delivering the medicine, ranging from a micro-needle skin patch to painless EpiPen-type administration, in order to make the dosing more convenient. De Vries hopes to expand the use of Ruconest to treat pre-eclampsia, a condition that affects 3% of women in pregnancy and for which there is no cure, and acute kidney injury — also known as contrast-induced nephropathy.