LONDON - Pharming Group NV will extend its Phase II study of transgenic alpha-glucosidase in the treatment of Pompe's disease following completion of the first six-month phase in which three infants who were expected to die survived, and showed significant improvement.

The children were in intensive care on respirators at the start of the treatment; two are now fit enough to attend the hospital on an outpatient basis while the third is being weaned off the respirator. Pompe's, an inherited, lethal muscle disease, usually results in death from cardiomegaly (enlargement of the heart).

A spokesman for Pharming, based in Leiden, the Netherlands, told BioWorld International, "The full biochemical data will be published in due course, but the significant point is that the children have reached an age that they would not have been expected to reach."

The juvenile arm of the trial, which is taking place at the Sophia Children's Hospital in Rotterdam, is now fully enrolled, and preliminary observations suggest the compound is well tolerated in this population.

The existing patients will continue to receive the treatment, and a second six-month trial will now commence, to which a further eight patients will be recruited.

The FDA has awarded Pharming Orphan Drug status for alpha-glucosidase, which is extracted from the milk of transgenic rabbits. The spokesman said that while it had planned to do a U.S. trial, the company is in "constant discussion" with the FDA to establish if the European data is so clear-cut that it could be accepted for U.S. registration. Pharming expects to be able to proceed with a European registration on the basis of the second six-month Phase II trial.

Pharming also said it opened its farm in Vienna, Wisconsin, which it says is the first in the world to raise transgenic cattle for the production of human proteins. Built in compliance with FDA recommendations, the farm cost US$4 million and houses 80 animals. Pharming produces the animals in collaboration with Infigen Inc., of DeForest, Wis., a specialist in nuclear transfer.

To date, 40 animals have been generated, all of which are in good health and display normal growth rates. Pharming says a significant number of these carry transgenes for the production of therapeutic proteins.