Ibrutinib receives FDA boost

Great news! Ibrutinib, the experimental drug that my wife, Mary Jo's, nephew has been taking as part of a clinical trial at Vanderbilt has taken another step towards general availability

Less than a year after the Food and Drug Administration began accepting applications for “breakthrough therapy” designations to fast-track promising new drugs, regulators have approved just 11 of 35 requests from pharmaceutical companies.

Three of those approvals, however, have gone to one niche market cancer drug that Raritan-based Janssen Pharmaceuticals is developing with Pharmacyclics Inc. of Sunnyvale, Calif.

The drug, called Ibrutinib, has so far proved effective at blocking three different types of blood cancers in patients who had relapsed several times after taking existing therapies. The FDA designations are for chronic lymphocytic leukemia, mantle cell lymphoma and Waldenstrom’s macroglobulinemia.

Industry analysts say Ibrutinib has mostly flown under the radar because of its small market, but still believe it holds significant potential for its unique technology and relatively mild side effects.

Last week, the FDA granted Ibrutinib its latest breakthrough designation to treat CLL, one of the most common forms of adult leukemia that affects about 16,000 Americans a year. The disease, a slow-growing cancer that starts in the white blood cells, targets the elderly, with an average survival of about five years.

Clinical trials have shown Ibrutinib is effective for all CLL patients but works particularly well in those with a rare form of the disease that makes them immune to chemotherapy treatment, said Adrian Wiestner, a senior investigator at the National Institutes of Health in Bethesda, Md., who is overseeing one of the studies. That 52-patient study has shown “dramatic improvement for patients who otherwise had run out of options,” Wiestner said.

The other two diseases that received FDA designation affect a smaller population of adults than CLL.

A breakthrough designation is intended to expedite the development and review of a drug for serious or life-threatening conditions, according to the FDA. The drug in development must show early clinical evidence that demonstrates substantial improvements over existing therapies.

A majority of patients who took part in Ibrutinib’s two clinical trial phases are still in treatment, up to two years later without relapse, said Craig Tendler, vice president of Janssen’s oncology late stage development. Janssen is a unit of New Brunswick-based Johnson & Johnson.

Results have not been released for its third and final clinical trial phase.

Taken in pill form — one a day — Ibrutinib is also more tolerable than traditional chemotherapy medicines, Tendler said. He attributes part of Ibrutinib’s success to “the selectivity of this drug over other available therapies.” Most traditional medicines, he added, are “a combination of chemotherapy regimens that are often toxic and have short remission. This keeps the disease under control. Its potential is really transformational.”

Read the entire article here:

Bad News Is Actually Good News

 I was to start the Ibrutinib trial soon specifically for Waldenstroms at Northwestern Univ. I got a call from the trial coordinator, she told me I have bad news, that is actually good news. The trial got canceled because of the "Breakthrough" status. Because "Breakthrough" is new, no one really knew what it meant.

Apparently it means Ibrutinib is being approved immediately in a limited way for CLL, Mantle Cell and Waldenstroms. The institutions that participated in the e arly Trial willbegin to prescribe Ibrutinib soon. It will soon be available to all institutions. This drug has been so successful, it is being rushed to market.

So instead of having to drive to downtown Chicago and go through the involved trial process, with CT scans BMB's etc. I can simply take the drug on a daily basis and get tested periodically.

 There are trials in process for the big ones as well, DLBCL, FNHL as a singleagent as well as in combination with Rituxin etc.

 Mark 55 Chicago