Out of Luck ... For Now

There has been a shortage of Fabrazyme — the medication used to treat Fabry's disease — for more than a year, as the maker, Genzyme, struggles with manufacturing problems, says Pharmalot's Ed Silverman. But when three patients petitioned the US Department of Health and Human Services to break Genzyme's patent so they could obtain a license to produce a new treatment, NIH denied the bid, saying such a move wouldn't help with the shortage problems in the near future, given the amount of time it takes to conduct clinical trials and gain FDA approval for new medications. Genzyme recently paid $175 million in fines for its manufacturing problems, Silverman says, and has promised to get the supply problem under control by next year. The three patients, having very little confidence that Genzyme would be able to restore the supply of Fabrazyme, contend that HHS has the power to override the patent because NIH paid for research at Mount Sinai School of Medicine, which licensed Fabrazyme to Genzyme, Silverman adds. If Genzyme is unable to stick to its timeline to have its manufacturing and supply problems sorted out by next year, however, NIH says it will allow other drugmakers to apply for licenses to produce another medication.