by David Wallace
Fedratinib by Celgene, offered on Expanded Access Program (EAP) for Myelofibrosis Patients
This is hot off the press! So hot, that no one has covered it until now – brought to you by PV Reporter.
I knew after combing through the ASH (American Society of Hematology) abstracts that something was missing. That important abstract that would provide a new glimmer of hope to so many myelofibrosis patients who have few treatment options. I was specifically looking for information on Celgene’s “second generation JAK2 inhibitor” Fedratinib.
There were a few abstracts, but nothing noteworthy. I was expecting to see good news for myelofibrosis patients who only have 1 FDA approved drug, Ruxolitinib (Jakafi).
A Quiet Hush from Drug Maker
Calls to Celgene’s drug information line requesting more information were met with a couple of “let me put you on hold” responses. Each time the agent came back, he had nothing new to report. He offered to escalate the issue and told me someone would call me back the next day. I told him, I would have the information before that. As it turned out, a Celgene representative called me back in short order. I introduced myself and told her about my discovery of their “yet un-publicized” Expanded Access Program (EAP) for Fedratinib for myelofibrosis patients. When I asked for more information, she said she could “only forward a request from a physician.” I said “you can’t really say anything…right?” She said correct.
A Reporter’s Hunch
Because I have been in this “MPN reporting business” for 5 years, my guess is this news will be released as a “late breaking abstract” at the ASH conference in San Diego. ASH is the world’s largest professional meeting concerned with the causes and treatment of blood disorders, hosting more than 25,000 attendees. These late breaking abstracts are usually significant enough to be added last minute for their high impact appeal and industry importance. At that time, we should have more details to go on.
Expanded Access Program Defined
Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics, and medical devices used to diagnose, monitor, or treat patients with serious diseases or conditions for which there are no comparable or satisfactory therapy options available outside of clinical trials. The US Food and Drug Administration (FDA) facilitates the expanded access process; however, access to investigational treatments requires not only FDA’s review and authorization but also the active involvement and cooperation of other parties, including drug companies and health care providers, in order to be successful.
This is exciting news, as it offers a second line treatment option to myelofibrosis patients who are not having optimal results with Jakafi.
January 4th, 2019 Update – Bristol-Myers Squibb to Acquire Celgene (CELG)
Bristol-Myers (BMY) Becomes a Cancer Fighting Giant with Celgene buyout. BMY has decided to buyout CELG in one of the healthcare sector’s biggest deals ever.
With the deal, Bristol-Myers eliminates many of its problems and becomes one of the biggest pharmaceutical stocks targeting cancer and oncology drugs. For Celgene, the deal represents one of the best outcomes for the struggling biotech. Look for more acquisitions in the healthcare space in 2019.