FDA Approves Drug for Adults with Rare Form of Bone Marrow Disorder

FDA has granted accelerated approval for Vonjo (pacritinib) capsules to treat adults with myelofibrosis, a rare form of bone marrow disorder, along with low platelet levels.

Myelofibrosis disrupts blood cell production and causes extensive bone marrow scarring, which can lead to severe anemia and a reduced number of platelets. This can increase the risk of bleeding and cause the spleen to enlarge. Myelofibrosis can be a primary or secondary disease, occurring on its own or developing due to another bone marrow disorder, respectively.

The effectiveness of pactirinib was based on the number of patients who had a 35% or greater spleen volume reduction from the start of the study to week 24. Twenty-nine percent of patients in the pactirinib treatment group experienced a spleen volume reduction of 35% or more, compared to the 3% in the standard treatment group.

Accelerated approval was granted on the condition that pactirinib must go through another study that confirms that spleen volume reduction leads to clinical benefits for the patients, such as improvements in function or feeling.

Source: FDA