In an interview with The ASCO Post following FDA’s recommendation that sales of ponatinib (Iclusig) be suspended, (see here) Brian J. Druker, MD, Director of Oregon Health & Science University Knight Cancer Institute and JELD-WEN Chair of Leukemia Research, had concerns about obtaining the drug for those patients who may respond and have no other treatment option.
Patients With T3151 Mutation
“Right now if a patient came in with a T3151 mutation, which doesn’t respond to any other medication, I don’t know that I can get that patient ponatinib. I haven’t seen any instructions or guidance about how to get access to this medication. I know that Ariad is working very hard with the FDA to come up with a plan but I haven’t seen anything yet,” said Dr. Druker.
In clinical trials conducted before ponatinib’s approval, serious blood clots occurred in 8% of patients taking the drug and blood clots in the veins occurred in 3% of patients. More recent data found approximately 24% of patients in the phase II clinical trial (median treatment duration, 1.3 years) and approximately 48% of patients in the phase I clinical trial (median duration, 2.7 years) experienced serious adverse vascular events. The events included life-threatening heart attack, stroke, loss of blood flow to the extremities resulting in tissue death, and severe narrowing of blood vessels in the extremities, heart, and brain requiring urgent surgical procedures to restore blood flow.
“We clearly have to take the FDA review quite seriously,” said Dr. Druker. “[But] I have to say that I have lots of patients who are doing extremely well on [ponatinib]. They had no other treatment options. I hope that we will be able to continue to treat them and should be able to treat other patients in a similar circumstance who have no other decent treatment options.” ■
The U.S. Food and Drug Administration (FDA) has asked the manufacturer of the tyrosine kinase inhibitor ponatinib (Iclusig) to suspend marketing and sales of the drug because of the risk of life-threatening blood clots and severe narrowing of blood vessels. Ariad Pharmaceuticals has agreed to...