The Food and Drug Administration has provided information on how health care professionals can ensure that patients who have benefitted from the leukemia drug ponatinib continue to have access to the treatment.
Less than a week after the agency announced that marketing and sales of the drug had been suspended because of the risk of life-threatening blood clots and severe narrowing of blood vessels associated with treatment, the FDA posted instructions on how to obtain emergency access to ponatinib through an Investigational New Drug (IND) application. This process entails contacting the FDA to obtain an emergency IND number for each patient who would benefit from continuing treatment and providing the manufacturer with that number to get ponatinib (Iclusig).
Health care professionals can obtain INDs for multiple patients during one phone call and should contact the agency at least 48-72 hours before the drug is needed, according to an FDA announcement.
The statement noted that health care professionals "may continue to use Iclusig for patients who they determine are responding to the drug and for whom the potential benefits outweigh the risks."
Ponatinib is a kinase inhibitor marketed by ARIAD Pharmaceuticals. It was approved in December 2012 for treating chronic myeloid leukemia and Philadelphia chromosome–positive acute lymphoblastic leukemia in adults.
Serious adverse events associated with ponatinib should be reported online to the FDA or by phone at 800-332-0178. Information about the IND program is available online. The FDA’s emergency IND telephone number is 301-796-7550 (between 8 a.m. and 4:30 p.m. EST, weekdays and 866-300-4374 after 4:30 p.m. EST. More information on how to obtain an IND for patients on ponatinib is available at http://www.fda.gov/Drugs/DrugSafety/ucm373040.htm.