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Interessante aanvulling, US ALS patienten zijn wakker aan het worden?
www.timesdispatch.com/servlet/Satelli...Insmed's iPlex studied in Italy for ALS treatment
TECH BUZZ
JEFFREY KELLEY Jan 20, 2007
Richmond-based Insmed Inc. has already cleared the hurdle of having its drug approved by federal regulators in the U.S.
But the company hasn't stopped there, setting its sights on the rest of the world to commercialize iPlex.
And Italy has come knocking on Insmed's door hoping to learn more about the drug's capabilities.
This month, the Italian Ministry of Health requested Insmed make its drug available to the country's physicians to study iPlex for treatment of Lou Gehrig's disease.
The fatal illness -- also known as amyotrophic lateral sclerosis, or ALS -- affects nerve cells in the brain and spinal cord and can eventually lead to paralysis before death. Most patients' minds remain unaffected, leaving them without motor skills but with the ability to recall everything before they were diagnosed.
But iPlex is approved and commercialized only in the U.S., and for a totally unrelated disorder: helping children who are abnormally short gain a few inches.
"This is a unique situation . . . a petition from a country to get their hands on a drug that isn't approved for the indication," Geoffrey Allan, Insmed's president and chief executive said last week. "That kind of testifies to the fact that [ALS] is a very debilitating disease."
Italy diagnoses about 1,000 new cases of ALS annually, according to Insmed, a publicly-held company considered one of the most well-regarded homegrown biotechnology firms, regional experts say.
So far, about 40 patients in Italy have expressed interest in iPlex for ALS, Allan said. The key ingredient in iPlex has protected motor neurons and promoted muscle and nerve regeneration in animals.
Italian doctors will use iPlex for tests only, and Insmed will be reimbursed through the country's health authorities for the expensive drug. Allan hopes these studies lead to clinical trials for iPlex use for ALS.
The news of possible treatment has sparked requests from Americans who suffer from ALS, though Insmed is unable to help -- for now.
"We will evaluate how to initiate a similar type program here," Allan said. "But at present we are not able to provide drugs for off-label use."
Insmed also is pursuing an application for iPlex approval in the European Union, which Allan said could be completed by midyear. If approved, it'll be next year before the company's drug is sold overseas -- likely through a commercial partner. Japan also is on the checklist of possible countries to approve and market iPlex.
But treating short stature, iPlex's primary use, won't be the big moneymaker for Insmed. In the longer-term, the company is hoping it will be used to treat more common disorders such as diabetes or muscular dystrophy.