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Cyclodextrin

FDA grants “Orphan Drug Designation” to Cyclodextrin for treatment of Niemann-Pick Disease Type C

Due to the efforts of Hugh and Chris Hempel, Dr. Caroline Hastings and Ron Browne, an application to the Food and Drug Administration requesting “Orphan Drug Status” for Cyclodextrin has been approved. 

What exactly does that mean for our Niemann-Pick Disease community? 

Here is a bit of background and glossary that will help you to understand this latest development:

Orphan Drug Designation

An Orphan designation is a status granted to a drug if it meets two criteria:

1.  The drug is intended to treat a rare disease or condition.  Rare = less than 200,000 patients in the US with this condition.

2.  The drug shows “promise” to be able to treat the condition.  Promise can mean that the drug appears to have an effect in a test tube or an animal model (“preclinical”) or there may be some data in people that shows it might work.  This is not an assessment of whether the drug is effective or not, or is safe or not, only that it may work.

If a drug meets both of these conditions, per the assessment and review of the information by the Office of Orphan Product Development (OOPD), then the drug will be given an Orphan designation.  An Orphan designation provides predominantly financial incentives to the developer as described in the Orphan Drug Act. 

For example, one financial benefit for an Orphan-designated drug is that the developer does not have to pay a user fee when the developer submits a marketing application.  It is important to emphasize that an Orphan designation does not make any assessment at all on how the drug works in clinical trials, whether it is safe or effective in patients, nor whether it will ever be commercially available – the Orphan designation’s main purpose is to make the development of the drug more financially viable. 

To receive an Orphan drug designation, there is no requirement for any hard data, and not even a requirement for human data.

The benefits to the developer receiving an Orphan Drug designation include:

1) marketing exclusivity, which is “defended by the FDA” (perhaps even “better than a patent”)

2) freedom from filing fees --it typically costs $1.4 million to file a New Drug Application (NDA) with the FDA

3) tax credits of 50% on the costs of the clinical trials

Glossary

FDA approval:  An FDA approval is referring to whether the drug has received a marketing approval.  That is, an approved drug would be commercially available and a licensed physician could prescribe the drug.  This is an entirely separate issue from the Orphan designation, which again, provides financial incentives for drug development.         

Investigational Treatment:  In the US, clinical investigations with experimental drugs can only be performed under Investigational New Drug applications (INDs).  An experimental drug is defined as a drug that has not received marketing approval from the FDA and is not commercially available for the intended use.  A clinical investigation is defined as any experiment in which a drug is given to one or more patients.  That is, any use of a drug except for the use of a marketed (approved) drug in the course of medical practice. 

The process an experimental drug, Orphan or non-Orphan, must go through in order to obtain a marketing approval is complex and highly variable depending on many, many things.  It would not be possible to make even a reasonable estimate of a timeline or process outline for Orphan drugs in general, because this varies so greatly depending on the specific circumstances. 

Additional Resources

There is additional information on the FDA Web site called FDA Basics that may be helpful in providing some background on drug development.     http://www.fda.gov/AboutFDA/Basics/ucm192696.htm

Click here for an article titled Drug Discovery, Development and Approval Process, outlining the process required for a drug to travel from the lab to patients in the U.S.

[May 21, 2010 mem]

“The National Niemann-Pick Disease Foundation (NNPDF) does not engage in the practice of medicine. It is not a medical authority nor does it claim to have medical knowledge. This site is an educational service of the National Niemann-Pick Disease Foundation and is not meant to provide diagnostic or treatment advice. Information contained or suggested on this Web site does not constitute medical advice. For all information related to care, medication or treatment, the NNPDF recommends consulting a physician to determine if information presented is applicable. Please review these additional cautions about medical information provided on the Internet.”