FDA Clarification On Orphan Status for Ponatinib

I wrote to the FDA to get clarification on pona's Orphan designation vs Aproval.  Here is their reply verbatim.

Orphan drug designation is a special status that is awarded to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor.  For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the orphan drug regulations at 21 CFR Part 316.  One of these conditions is that the sponsor must submit the request for orphan drug designation prior to the application seeking marketing approval for that particular use.  The drug may have received marketing approval for other uses, including uses that are not to treat a rare condition or disease.

In terms of ponatinib for use in the treatment of gastrointestinal stromal tumors (GIST), the product has achieved designation status but has not received FDA marketing approval for that use.  Ariad cannot market ponatinib for the GIST orphan use unless they submit a marketing application for the drug and it is approved.

I hope this provides clarification.

[Signed]

[xxxxxxxxx], R.Ph.
Health Science Administrator
Office of Orphan Products Development
Food and Drug Administration