TEL AVIV, July 23 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
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.