Science & Enterprise subscription

Follow us on Twitter

  • A new enterprise in the U.K. is creating treatments with stem cells to repair damaged nerve cells in the inner ear… https://t.co/y4qOmPcuWK
    about 3 hours ago
  • New post on Science and Enterprise: Start-Up to Use Stem Cells for Hearing Loss https://t.co/dVEfRVHeTi #Science #Business
    about 3 hours ago
  • The U.S. Air Force is funding research at Massachusetts Institute of Technology to speed development of artificial… https://t.co/zbW38MuPMP
    about 6 hours ago
  • New post on Science and Enterprise: MIT, Air Force to Open A.I. Accelerator Lab https://t.co/tj0PbSXL9h #Science #Business
    about 6 hours ago
  • A solid majority of Americans express at least some doubts about biometrics for accessing computers, this weekend's… https://t.co/oUU1W1bX6E
    about 2 days ago

Please share Science & Enterprise

RSS
Follow by Email
Facebook
Facebook
Google+
Twitter
Visit Us
LinkedIn
INSTAGRAM

Treatment for Rare Bone Marrow Disease Gains FDA Approval

Jakafi bottles (Incyte Corp.)

(Incyte Corp.)

The Food and Drug Administration today approved the drug ruxolitinib, to treat the bone marrow disease myelofibrosis, for marketing in the U.S. Ruxolitinib is marketed under the brand name Jakafi by Incyte Corporation, a biotechnology company in Wilmington, Delaware.

Jakafi is a pill taken two times a day to inhibit enzymes called Janus Associated Kinase (JAK) 1 and 2 involved in regulating blood and immunological functioning. The disease myelofibrosis is associated with the deregulation of JAK 1 and 2, in which bone marrow is replaced by scar tissue resulting in blood cells being made in organs such as the liver and the spleen.

The disease is characterized by an enlarged spleen, anemia, and decreased white blood cells and platelets. Symptoms include fatigue, abdominal discomfort, pain under the ribs, feeling full (satiety), muscle and bone pain, itching, and night sweats.

FDA approval was based on results from two randomized phase 3 trials (COMFORT-I and COMFORT-II) that showed patients treated with Jakafi experienced significant reductions in splenomegaly (enlarged spleen). The COMFORT-I trial, conducted by Incyte, compared Jakafi to placebo in 309 patients with primary myelofibrosis, or myelofibrosis symptoms resulting from related disorders. The COMFORT-II trial, conducted by Novartis, Incyte’s partner outside of the U.S., compared Jakafi to best available therapy — either hydroxyurea, a chemotherapy agent, or glucocorticoids — in 219 patients.

A greater percentage of patients receiving Jakafi, says FDA, experienced more than a 35 percent reduction in spleen size when compared to patients receiving placebo or best available therapy. A greater proportion of patients receiving Jakafi also saw more than a 50 percent reduction in their myelofibrosis-related symptoms, including abdominal discomfort, night sweats, itching, and bone or muscle pain, than was the case in patients receiving placebo.

FDA says it reviewed Jakafi under the agency’s priority review program, an expedited six-month review of drugs that may offer significant advances in treatment over available therapy or that provide a treatment when no adequate therapy exists. Jakafi was also designated as an orphan drug, which treats a disease affecting fewer than 200,000 people in the U.S.

Read more:

*     *     *

Please share Science & Enterprise ...
error

Comments are closed.