The FDA grants orphan drug status to incentivize the development of one of a kind drugs and other medical treatments. This status grants the company that manufactures the drug marketing exclusivity for 7 years from the date of receipt of marketing approval of the drug. The recognition of orphan drug status also enables accelerated examination and response paths from the FDA and other regulatory authorities.
The granting of orphan drug status for the AstroRx® product is an acknowledgment of the uniqueness and medical potential of the product and will give a significant boost to its entry into the market once it's approved for marketing.
Kadimastem is progressing in the Phase 1/2a clinical trial in the Hadassah Ein Karem Hospital. The company completed enrollment of 5 patients for Cohort A and began cell transplantation in the patients. Results for Cohort A of the trial are expected in mid-2019.
AstroRx® is a unique, first in human, cell therapy product for ALS. The product consists of nerve-supporting cells (astrocytes), which are produced at Kadimastem from pluripotent stem cells with unique technology developed by the company. Treatment is administered by a simple injection into the spinal cord fluid of ALS patients, the advantage of AstroRx® is to replace the poor functioning of the supporting cells in the patient, with healthy cells.
Mr. Yossi Ben-Yosef, CEO of the company, said: "I am proud that the medical product in which we are conducting a clinical trial today, AstroRx®, has received orphan drug status. The FDA has provided further confirmation of the uniqueness and great medical potential of the product. I believe that the approval will increase the possibility of reaching the market faster and will provide significant advantages in marketing the product."
Prof. Michel Revel, Kadimastem's Chief Scientist, commented: "The FDA's decision is a recognition of the uniqueness and excellence of the Kadimastem's flagship product, which is in a clinical trial for the treatment of the severe neurological disease ALS. This recognition is a badge of honor for the company and will help accelerate the delivery of AstroRx® cells for use in ALS patients worldwide."
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