AURORA, Colo.--(BUSINESS WIRE)--The Food and Drug Administration has given Taiga Biotechnologies Inc. Orphan Drug Designation for an experimental product designed to treat children with a rare immune deficiency that forces them to live in a sterile environment.
“The ultimate goal of our approach is to see an improvement of stem cell engraftment and a decrease in recovery time for these patients,” said Yosef Refaeli, CEO of Taiga, headquartered in Aurora, Colo.
“This is a key regulatory milestone that comes with a number of benefits, including seven years of marketing exclusivity, tax credits, and regulatory support from the Office of Orphan Product Development,” said Brian Turner, president and Chief Scientific Officer of Taiga.
Taiga was given Orphan Drug Designation, for its first clinical product called TBX-1400, by the Office of Orphan Products Development of the FDA on June 2nd, 2016.
Federal law allows granting of this special status for drugs used to treat rare diseases or conditions upon request of a sponsor. To qualify, both the drug and the disease or condition must meet certain criteria. Companies have relied on this program in the past to improve the likelihood of successful commercial development by leveraging the various governmental financial incentives and regulatory support for these types of eligible products.
The product will be tested in children who suffer from Severe Combined ImmunoDeficiency, known more commonly as SCID or “bubble baby” disease.
These patients must live in a sterile environment because they do not have a functioning immune system.
Without a bone marrow transplant these children will eventually die from common infections.
“We believe that by adding our proprietary protein to the blood stem cells prior to transplant we will improve the outcome of the procedure by reducing the time it takes for the stem cells to develop into a new immune system,” Refaeli said. “This could dramatically reduce the amount of time a patient must spend in the hospital and hopefully be a critical component to every bone marrow transplant procedure in the near future.”
The clinical trial will take place in two FDA-approved medical centers in Israel: Schneider Children’s Hospital in Petach Tikvah and Hadassah Ein Kerem in Jerusalem. By working overseas, Taiga believes it can reduce the cost of the clinical trial and have better access to patients afflicted with this rare genetic disorder. There are small pockets of people living in Israel where the disease is much more prevalent than in the general population.
Severe combined immunodeficiency (SCID) is a life-threatening primary immunodeficiency disease caused by defects compromising the quantity and function of specialized immune cells called T-cells and B-cells. Infants with SCID suffer from repeated life-threatening infections that usually lead to a diagnosis by the age of three to six months. Left untreated, children continue to experience severe infections; this usually leads to death by 2 years of age. Children with SCID are currently treated using bone marrow transplantation to reconstitute a normal immune system. Outcomes of these transplants are best for the youngest infants, who are transplanted before they have suffered from a severe infection (most of these infants are either siblings of a proband or are identified by neonatal screening programs), and for patients with a matching family donor. In contrast, the results of transplants performed on older infants and those performed with cells from alternative donors are less favorable.
About Taiga Biotechnologies, Inc.
Taiga Biotechnologies, Inc. is a Colorado-based biotechnology company focused on the development of novel therapies for complex diseases such as hematological conditions, cancer, and infectious diseases such as HIV and Influenza. TBX-1400 is one of Taiga’s leading development programs about to enter into clinical development. For more information, please visit: www.taigabiotech.com.
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