Tag Archives: global cancer drug

FDA announces orphan drug designation for DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa)

Dimension Therapeutics Inc., a biopharmaceutical company specialized in developing gene therapies targeting liver related diseases, declared that US Food and Drug Association (FDA) granted orphan drug status for DTX401 for the treatment of patients with Glycogen Storage Disease Type Ia (GSDIa). DTX401 is the first AAV gene therapy to enter IND-enabling studies for the treatment of GSDIa.

DTX401 has been designed to serve underlying genetic effect for which it delivers stable expression and activity of the enzyme glucose-6-phosphatase (G6Pase). It improves G6Pase activity and reduces hepatic glycogen levels, a well-described biomarker of disease progression, in multiple in vivo preclinical models.

FDA provides a number of benefits to the drug developers or pharmaceutical companies through development and commercialization. Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Firdapse gets orphan drug status for treatment of Myasthenia Gravis

Catalyst pharmaceuticals Inc., a biopharmaceutical company, declare its novel therapy Firdapse (amifampridine phosphate) as orphan drug for the treatment of patients with Myasthenia Gravis, as approved by Food and Drug Association (FDA). The company focuses on developing and commercializing new therapies for the treatment of rare debilitating diseases.

Myasthenia Gravis is the rare neuromuscular disease antibodies to the muscle-specific kinase (MuSK-MG) which inflicts approximately 5-8% of Myasthenia Gravis patients which is equal to 4,500 patients in the US. The characteristics of myasthenia gravis are predominance in women, prominent bulbar involvement, sever clinical conditions and resistant to treatment. Presently, there are anticholinesterase inhibitors or immunosuppressant available for the treatment of the disease.

FDA provides a number of benefits to the drug developers or pharmaceutical companies through development and commercialization. Orphan Drug designation is granted by the FDA’s Office of Orphan Products Development for drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Orphan Drug designation qualifies a company for several benefits under the Orphan Drug Act 1983. These benefits include assistance with clinical study design in drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

FDA grants VX15 from Vaccinex Inc. Orphan Drug designation for Huntington’s disease

VX15 developed by Vaccinex Inc., a pharmaceutical company focusing on the discovery and development of therapeutic monoclonal antibodies to treat patients with cancer and neurodegenerative diseases, has been designated as orphan drug by Food and Drug Association (FDA) for the treatment of Huntington’s disease.

Huntington’s disease (HD) is a hereditary neurodegenerative disease that causes progressive breakdown of nerve cells in the brain. The disease is commonly found in middle aged people followed by severely affecting them in next 5-10 years.

FDA grants orphan drug status for rare and life threatening diseases that affect less than 200,000 people per year in the U.S. Also, FDA provide several beneficiaries to drug developers including assistance with clinical study design and drug development, tax credits for qualified clinical trials costs, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory product approval.

 

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com

Coversin by Akari therapeutics receives Orphan drug Designation from FDA

Coversin developed by Akari therapeutics, an emerging pharmaceuticals company dedicated to the development and commercialization of innovative therapeutics to treat orphan autoimmune and inflammatory diseases has been granted orphan drug designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria.

PNH is an ultra-rare, life-threatening and debilitating disease of the blood with estimated 8,000-10,000 patients across North America and Europe. The PNH patients suffer from a certain genetic deficiency in which they allow their own complement system to attack and destroy blood cells, leading to life-threatening complications.

According to FDA, orphan drug designation status is given to those drugs or treatments which promise to cure or prevent rare life threatening diseases. By FDA, rare diseases are defined as those which affect less than 200,000 people in US. Also, FDA helps the companies which cultivate such drugs by providing them with incentives to sponsor developing drugs and biologics. The receipt of Orphan Drug Designation status does not change the regulatory requirements or process for obtaining marketing approval and designation does not mean that marketing approval will be received.

 

For Orphan Drug Clinical Insight Reports Contact:  neeraj@kuickresearch.com