US FDA Grants Orphan-Drug Designation for ViroMed’s ‘VM202-ALS’ for Treatment of Amyotrophic Lateral Sclerosis (Lou Gehrig’s Disease)
On Feb. 14th, biopharmaceutical development company ViroMed Co., Ltd. (CEO Kim Yong Soo) announced that the US FDA granted ViroMed’s ‘VM202-ALS’ for treatment of amyotrophic lateral sclerosis (Lou Gehrig’s disease) the orphan-drug designation.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis, or Lou Gehrig’s disease, is a neurodegenerative disease of unknown causes where the motor neurons necessary to move muscles in our body are destroyed, leading to paralyzation of all muscles such as in tongue, neck, and the limbs. The disease occurs mostly in adults and within 2-5 years of symptoms onset, most will die due to respiratory failure. ALS occurs in 3~8 out of 100,000 people and there are currently an estimate of 400,000 patients around the world. Roughly 20,000 ~ 30,000 patients are in the US alone. 5,000 new patients are diagnosed with the diseased every year in the US (NIH, Amyotrophic Lateral Sclerosis Fact Sheet).
If phase II clinical study of VM202-ALS is successfully completed in the US, early commercialization of the product by FDA’s priority review for NDA approval can be possible. These expedited processes will significantly cut down development cost and period of the drug. In addition, the orphan-drug designation also allows for 7 years of market exclusivity even after expiration of related patents.
VM202 is a DNA based drug that expresses a protein called HGF when intramuscularly injected into the patient. This protein is responsible for inducing angiogenesis (formation of new blood vessels) and regeneration of damaged nerve cells, which are the primary functions exploited.
VM202 is currently being developed in clinical stages for ischemic cardiovascular disease and neurodegenerative diseases. Phase II clinical study in the US for VM202-PAD was successfully completed in Nov. 2013, while 149/200 patients were enrolled for VM202-PAD phase II clinical study in China. Phase II clinical study for VM202-DPN is currently in the follow-up stage to be completed in March with results expected by the end of September of this year. Phase I clinical study for VM202-CAD has been completed in Korea and phase II clinical study is planned for 4Q of this year.
ViroMed received approval from the US FDA in Oct. 2013 for phase I/II clinical study of VM202-ALS. The study will be conducted at Northwestern University Hospital where ALS center is already established.