TEL AVIV, July 23 (Reuters) - Pluristem Therapeutics Inc will apply to the U.S. Food and Drug Administration for approval of its placenta-based stem cell treatment for aplastic bone marrow as an orphan drug or rare disease treatment, it said on Monday.
Gaining orphan drug status is part of Pluristem’s strategy for penetrating the bone marrow recovery market, starting with treatment of aplastic anaemia, a disease that affects five to 10 people in every million.
Treatment costs range from $150,000 to $200,000 per person for the 60,000 bone marrow patients worldwide each year, though the majority of this is for hospitalisation. Pluristem said its treatment helps reduce hospitalisation time.
Orphan drug status has several potential benefits, including the possibility of an expedited regulatory process, availability of grant money, tax credits and seven years of market exclusivity.
In 2011, Pluristem received orphan drug status for its cell therapy in the treatment of Buerger’s disease, a rare blood vessel disease.
Pluristem said on May 9 that its cells had saved the life of a seven-year-old girl suffering from aplastic bone marrow and who had undergone two failed bone marrow transplants. Its stock rose 32 percent.
“Orphan drug status in the U.S. would help accelerate our path to full FDA approval and we intend to apply for a similar designation in Europe and global territories,” said Zami Aberman, chairman and CEO of Pluristem.