SC therapy for Stargardt's disease designated Orphan Drug

[barbara]

"The Scientist.com"
3-2-10

The US Food and Drug Administration (FDA) granted Advanced Cell Technology's (ACT) embryonic stem cell-based therapy for Stargardt's Disease the coveted title of being an Orphan Drug -- a designation aimed to promote "the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions," according to the FDA's website.

With this new status, ACT's therapy is eligible for tax credits, access to certain funding for clinical trials, and marketing exclusivity for as long as seven years after it is approved. The company says it plans to use the new orphan designation "to accelerate clinical development" of the therapy, which has yet to enter human trials.

From the FDA website:

Developing Products for Rare Diseases & Conditions

The Food and Drug Administration has charged The Office of Orphan Products Development (OOPD) to dedicate its mission to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD interacts with the medical and research communities, professional organizations, academia, governmental agencies, and the pharmaceutical industry, as well as rare disease groups.

The OOPD administers the major provisions of the Orphan Drug Act 1(ODA) which provide incentives for sponsors to develop products for rare diseases. The ODA has been very successful - more than 200 drugs and biological products for rare diseases have been brought to market since 1983. In contrast, the decade prior to 1983 saw fewer than ten such products come to market.

In addition, the OOPD administers the Orphan Products Grants Program2 which provides funding for clinical research in rare diseases. The Office Of Orphan Products Development will also administer a new grant program, the Pediatric Device Consortia (PDC) Grant Program3. The PDC Grant Program solicits grant applications from institution/organizations that propose to develop nonprofit consortia to facilitate pediatric medical device development. FDA will provide grants to consortia whose business model and approach to device development will either result in, or substantially contribute to, market approval of medical devices designed specifically for use in children. Although administered by the OOPD, this grant program is intended to encompass devices used in all pediatric diseases not just rare diseases.