Tag Archives: global orphand rug market

Iomab-B receives EMA orphan drug status for AML

Iomab-B manufactured by Actinium Pharmaceuticals Inc. has been declared as orphan drug by European Medicines Agency for the treatment of patients with Acute Myeloid Leukemia (AML). The drug has the clinical tested usage in the treatment of patients with relapsed or refractory Acute Myeloid Leukemia (AML) with an age crossing 55 years. The major use of the drug is to prepare patients for bone marrow transplant also called as hematopoietic stem cell transplant (HSCT). Currently, it is undergoing phase III clinical trial with 150 patients in the US.

The EMA grants orphan designation to rare diseases that are defined as life-threatening or chronically debilitating conditions that affect no more than 5 in 10,000 people in the EU. With an estimated 30 million people living in the EU this equates to approximately 250,000 people or less for each rare disease.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020

 

SGT-001 declared orphan drug for DMD

Solid Biosciences and Solid GT announces that US Food and Drug Administration (FDA) have granted Orphan drug status to SGT001 for the treatment of Duchenne Muscular Dystrophy (DMD). It is a rare and progressive muscle wasting disease that is usually diagnosed in childhood. SGT001 is the gene therapy mediated to amino-associated viral (AAV) vector. The gene therapy functions by potentially restoring the dystrophin expression in the muscles.

The drug has proved to provide long term systemic expression of comparatively shorter form of dystrophin in the muscles by its clinical studies. The gene therapy also benefits as improved muscle power and prevention from contraction induced damage. The therapy is expected to commercialize in early 2017.

The FDA Orphan Drug Designation is granted to medicines and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment medicine.

For Free Sample of Global Orphan Drug Clinical Pipeline Reports Contact: avinash@kuickresearch.com

Or Visit:  Global Orphan Drug Market Future Outlook 2020