November 16, 2011, 6:12 pm
F.D.A. Approves Drug for Bone Marrow Disease
Comment:Although researchers from the Mayo Clinic who helped test the drug in early clinical trials wrote in a letter to The New England Journal of Medicine last month that the drug’s effect on reducing spleen size was “modest and not always durable’’ and that patients could suffer severe effects when they stopped taking the drug. This drug was still approved and the drug reps will now be out pushing it to the doctors with a spin on the results using relative statistics…… the insanity in the medical world just gets worse.
Read the article, would you take this drug???By ANDREW POLLACK
The Food and Drug Administration on Wednesday approved the first drug to treat myelofibrosis, a rare but potentially life-threatening bone marrow disorder.
The drug, called Jakafi, is the first product to reach the market from Incyte, a biotechnology company based in Wilmington, Del. It is also the first approval for a class of drugs known as JAK inhibitors, which may be useful in treating a variety of cancers and inflammatory diseases. Pfizer is hoping to win approval as early as next year of a JAK inhibitor, tofacitinib, that some analysts think could be a blockbuster oral drug for treating rheumatoid arthritis.
Myelofibrosis is characterized by the buildup of abnormal blood cells and fibers and scarring in the bone marrow, which interferes with production of blood cells and can lead to enlarged livers and spleens. Symptoms can include anemia, fatigue, severe itching, night sweats and pain.
Incyte estimates that about 16,000 to 18,000 Americans have the disease. Typical survival is about five years, though some people live much longer. While there has been no specifically approved drug until now, patients can be treated with blood transfusions to combat anemia, certain chemotherapy drugs or bone marrow transplants.
Jakafi, also known as ruxolitinib, is a pill taken twice a day. Incyte said the price will be $7,000 for a 30-day supply, or about $85,000 a year. This was higher than some Wall Street estimates.
In a call with securities analysts, Incyte executives asserted that this was in line with pricing for other oral drugs for rare cancers. They said they expected insurers to pay for it. The company, as is now par for the course, will also offer co-payment assistance to reduce out-of-pocket costs that could deter some patients from using the drug.
In two clinical trials, Jakafi helped to shrink spleens and reduce symptoms, according to the data on the label. “In many patients, it’s transforming – people start enjoying life again,’’ said Dr. Srdan Verstovsek, a leukemia specialist at the M.D. Anderson Cancer Center who was the lead investigator in one of the clinical trials, said in an interview. Still, he said, while the drug was good in reducing spleen size and symptoms, it did not effectively treat other problems from the disease, like anemia.
In one clinical trial, 41.9 percent of patients getting Jakafi – compared to 0.7 percent getting a placebo — experienced a reduction of at least 35 percent in the size of their spleen after 24 weeks. Some 45.9 percent of the patients taking the drug reported at least a 50 percent reduction in symptoms compared to 5.3 percent of those getting the placebo.
In a second trial, 28.5 percent of patients getting the drug had a 35 percent or greater shrinkage in spleen size, compared to zero patients getting the best available therapy, as chosen by their physicians. This was usually a chemotherapy drug or a steroid.
The company has also said there is data showing that Jakafi prolongs survival, which will be presented at the American Society of Hematology meeting in December.
The main side effects include low platelet levels, anemia, fatigue, diarrhea and shortness of breath, according to the F.D.A.
Researchers from the Mayo Clinic who helped test the drug in early clinical trials wrote in a letter to The New England Journal of Medicine last month that the drug’s effect on reducing spleen size was “modest and not always durable’’ and that patients could suffer severe effects when they stopped taking the drug.
Eric Schmidt, analyst at Cowen & Company, estimated sales in the United States could exceed $400 million in 2016. Novartis has the rights to market the drug outside the United States.
JAK inhibitors block the action of proteins called Janus-associated kinases which are involved in signaling in cells. An abnormality in this signaling contributes to myelofibrosis.
“Jakafi represents another example of an increasing trend in oncology where a detailed scientific understanding of the mechanisms of a disease allows a drug to be directed toward specific molecular pathways,’’ Dr. Richard Pazdur, the head of cancer drugs for the F.D.A., said in a statement.
The F.D.A. approval was expected but came before the agency’s Dec. 3 deadline. Incyte shares rose 34 cents to $12.94 on Wednesday.