Early testing of drugs for rare diseases
A new National Institutes of Health (NIH) program in the USA, known as Therapeutics for Rare and Neglected Diseases, is funding and doing early testing on promising candidate drugs that was previously left to commercial interests to make happen.
The program falls under the National Centre for Advancing Translational Sciences unit within the NIH.
Most start-up drug companies need to raise significant funds to pay for all the tests necessary just to get permission to try their medicine in humans. But Indianapolis-based Chondrial Therapeutics LLC might have found a better way: it got the federal government to do the testing for it.
Chondrial, which is developing a drug for a rare condition known as Friedreich’s ataxia (a “cousin” of HSP) will announce Wednesday that it has been accepted into a program run by the National Institutes of Health in which the NIH’s own researchers conduct tests on Chondrial’s drug and pay for outside contractors as needed.
Those services are worth at least $5 million and possibly twice that much, said Steve Plump, CEO of Chondrial.
“It helps validate our scientific platform. They’ve looked at everything from our [intellectual property] to our manufacturing,” said Plump, who was formerly chief marketing officer at Indianapolis-based Eli Lilly and Co. “For Chondrial, it’s a dramatic expansion of our drug development expertise. [NIH researchers] actually are an extension of our company now.”
Chondrial’s goal is to apply with the U.S. Food and Drug Administration as early April to get approval for human testing of its drug.
SOURCE: Indianapolis Business Journal January 13, 2016 http://www.ibj.com/articles/56607
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