Investigational new drug means a new drug or biological drug that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes. The terms investigational drug and investigational new drug are deemed to be synonymous for purposes of this part.b) The availability for public disclosure of all data and information in an investigational new drug application for a new drug will be handled in accordance with the provisions established in 314.430 for the confidentiality of data and information in applications submitted in part 314.
Pulmonary arterial hypertension (PAH) is a rare, progressive disorder characterized by high blood pressure (hypertension) in the arteries of the lungs (pulmonary artery) for no apparent reason. The pulmonary arteries are the blood vessels that carry blood from the right side of the heart through the lungs.
Currently, there are 32 orphan drugs designated for treatment some aspect of sickle cell disease. Only hydroxyurea has orphan drug approval to reduce the frequency of painful crises and the need for blood transfusions in adult patients with sickle cell anemia with recurrent moderate to severe painful crises.That was approved in 1998.ickle cell disease is a rare genetic condition that affects 90,000 to 100,000 Americans, mostly African Americans.