Advanced Cell Technologies (ACT) just announced that their stem cell line used to treat Stargardt’s Macular Dystrophy has just been granted “orphan drug designation” by the FDA.
This means that ACT will “receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of as long as seven years.” In other words, the research will move faster and be more profitable for the company.
Orphan drugs or orphan diseases or those which affect less than 200,000 Americans and thus are often not profitable for research. This help from the federal government gives companies like ACT the incentive to study and hopefully one day cure rare diseases.
Interestingly, this is the first time a stem cell line has been granted orphan drug status.
Hello,
My name is Kelli and my husband Brian was diagnosed with Stargardts seven years ago. We banked our girls cord blood when they were born, so we are interested in knowing if this or any other gene therapy can help restore some of my husband’s vision. We are looking for cutting edge info treatments and clinical trials. Thank you ever so much for your time and assistance. Kelli
So glad you will have the exclusive rights to market for the next seven years. So with federal grant funding, I forsee financial blessings. Now there is some incentive.