According to a story from BioSpace, the biotechnology company Partner Therapeutics, Inc., recently announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to the company’s product sargramostim, marketed at Leukine. Leukine was first approved for public use in 1991 and has gained the designation as a therapy for pulmonary alveolar proteinosis. Partner is primarily focused on the development of therapies the improve cancer treatment outcomes.
About Pulmonary Alveolar Proteinosis
Pulmonary alveolar proteinosis (PAP) is a rare disorder of the lung which is characterized by the buildup of lipoprotein compounds in the alveoli, spaces in the lung which play a central role in gas exchange and ventilation. There are a number of potential causes of this illness. These include exposure to dust or chemicals, as a complication of certain cancers, or due to lung infections. However, the most common cause is autoimmune; this autoimmunity causes macrophages (a type of white blood cell) that normally are present in the lung to be absent. Symptoms include shortness of breath, fever, cough, and weight loss. The course of the disease in unpredictable; some patients may see their disease progress whereas others may see their illness go away on its own. Lung transplant or whole lung lavage are common treatments. To learn more about pulmonary alveolar proteinosis, click here.
About Orphan Drug Designation
Orphan Drug designation is typically reserved for therapies that are intended to treat diseases that are considered rare. In the US, this is defined as any disease that affects less than 200,000 people. In addition, the drug must offer advantages in either effectiveness or safety over currently available treatments, or must fulfill a currently unmet medical need. Orphan Drug designation confers a number of benefits to the recipient company. These include the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug is approved for public use.
About Leukine
Leukine is currently approved for a total of six different indications, including an approval earlier this year for acute radiation syndrome. It functions by stimulating the production of a variety of different white blood cells and promoting their functionality.
