Experimental Treatment for Myelofibrosis Earns Orphan Drug Designation

According to a story from proactiveinvestor.com.au, the drug company Pharmaxis Ltd. announced recently that the US Food and Drug Administration (FDA) has granted it Orphan Drug designation. This designation is in regards to the company’s investigational therapy candidate PXS-5505, which is being developed as a treatment for myelofibrosis, a rare cancer that affects the bone marrow. The drug is classified as a pan LOX inhibitor.

About Myelofibrosis

Myelofibrosis is considered a rare type of bone marrow cancer. The disease is characterized by the excessive accumulation of abnormal stem cells in the bone marrow which trigger a process called fibrosis, or scarring. Over time, the bone marrow is replaced with scar tissue. While the exact cause of myelofibrosis is not known, genetic mutations affecting the MPL, JAK2, and CALR genes are known risk factors. Symptoms of myelofibrosis include enlarged spleen, anemia, shortness of breath, easy bruising and bleeding, greater risk of infection, bone pain, gout, fatigue, weight and appetite loss, and increased blood cell volume. As a cancer that affects stem cells, stem cell transplant can cure the disease. However, this process carries many significant risks. Other forms of treatment are symptomatic and supportive and do not alter the course of myelofibrosis. There is a dire need for safer and more effective therapies for the disease. To learn more about myelofibrosis, click here.

About Orphan Drug Designation

Orphan Drug designation is generally reserved for therapies that are in development for diseases considered rare; this is defined as any illness that affects less than 200,000 people in the US. To qualify, a therapy must display advantages in either safety or effectiveness over currently available treatments. Alternatively, it must satisfy a medical need that is not being met by any current medications. The designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is approved for the market by the FDA.

PXS-5505 has undergone early trial testing for safety measures and has also demonstrated its potential capability to reduce fibrosis of bone marrow in two different models of myelofibrosis. The company is currently developing its plans for further clinical trials.


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