US approves first drug for bone marrow disorder

Pres­i­dent Barack Obama speaks before he signs an exec­u­tive order direct­ing the Food and Drug Admin­is­tra­tion to take steps to reduce drug short­ages. Also pic­tured are Health and Human Ser­vices Sec­re­tary Kath­leen Sebe­lius, sec­ond left, FDA Com­mis­sioner Peggy Ham­burg, right, phar­macy man­ager Bon­nie Fraw­ley from Brigham and Women’s Hos­pi­tal in Boston, Mass., left, and can­cer patient Jay Cue­tara from San Fran­cisco. (Asso­ci­ated Press | Charles Dharapak)

MATTHEW PERRONE

AP Health Writer

WASHINGTON — The first drug to treat a rare dis­or­der that causes red blood cells to build up inside bone mar­row was cleared Wednes­day by the U.S. Food and Drug Administration.

The FDA approved the twice-a-day pill Jakafi from Incyte Corp. to treat myelofi­bro­sis, which causes ane­mia, fatigue, pain and swelling of the spleen. The dis­ease spurs abnor­mal blood cells to build up in bone mar­row, form­ing thick scar tis­sue that slows the pro­duc­tion of healthy blood cells. To make up for the short­age, other organs includ­ing the liver and the spleen begin pro­duc­ing blood cells.

Incyte esti­mates the dis­ease affects between 16,000 and 18,500 peo­ple in the United States, although pre­cise fig­ures are not avail­able. The dis­ease cur­rently is treated with chemother­apy or bone mar­row trans­plant, and some patients are not eli­gi­ble for the procedures.

Incyte said it plans to launch Jakafi nation­wide next week through spe­cialty pharmacies.

Known gener­i­cally as rux­oli­tinib, Jakafi is the first drug to reach the mar­ket from the Delaware-based Incyte Cor­po­ra­tion. The com­pany has part­nered with Swiss drug maker Novar­tis, which holds the rights to mar­ket Jakafi abroad. The com­pany said Wednes­day that Jakafi will cost $7,000 per month, or $84,000 for a year’s sup­ply for insured patients. Incyte plans to pro­vide the drug free to unin­sured patients and will offer copay assis­tance to patients with finan­cial need.

The FDA approved the drug based on two stud­ies includ­ing 528 patients with the dis­ease. Patients were ran­domly assigned to receive a placebo or Jakafi. More patients in the drug group saw a sig­nif­i­cant reduc­tion in the size of their spleen as well as a 50 per­cent decrease in symp­toms, includ­ing pain, dis­com­fort and night sweats.

The drug works by block­ing two enzymes asso­ci­ated with the disease.

The FDA reviewed Jakafi under its pri­or­ity review pro­gram for impor­tant new ther­a­pies, which aims to clear drugs in six months instead of the usual 10.

Side effects reported in patients tak­ing the drug included diar­rhea, headache, dizzi­ness and nausea.

Posted by AP News on Nov 16 2011. You can follow any responses to this entry through the RSS Feed. Comments can be made below.