Today, Atara Biotherapeutics, Inc. announced that the the U.S. Food and Drug Administration (FDA) has granted ATA230 with a Rare Pediatric Disease Designation for treating congenital cytomegalovirus (CMV) infection.
CMV is a member of the herpes virus family, and is fairly common in adults. As a result, it can get passed along to babies in utero
A CMV is generally not a cause for concern in healthy people, but in babies and people with compromised immune systems, the infection can be life threatening.
Most babies with congential CMV won’t show symptoms of CMV infection after birth. In rare cases, however, symptoms may appear and can include premature delivery, jaundice, enlarged liver and spleen, microcephaly (small head), and feeding difficulties. These babies are also at risk for hearing, vision, neurologic, and developmental problems.
ATA230 is a T-cell immunotherapy that targets antigens expressed by CMV. Atara is developing several drugs that use cytotoxic T lymphocytes (CTL) to detect and kill abnormal or diseased cells in the body. In the case of ATA230, the approach seems to be working. Last December, at the American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego, CA, Susan Prockop, M.D., of Memorial Sloan Kettering Cancer Center, New York, NY presented results
of a Phase 2 study showing ATA230 was effective in 73.3% of the patients (11 of 15 patients) at high risk for developing a CMV infection.
To date, ATA230 has not been tested in children.
: “FDA’s Rare Pediatric Disease Designation, following the recent orphan drug designation for ATA230, further underscores the high unmet medical need in treating congenital CMV infection,” said Isaac Ciechanover, M.D., Chief Executive Officer and President of Atara Biotherapeutics. “We will continue to work closely with FDA and other global health authorities to evaluate the development of ATA230 to address this potentially life-threating disease.”
The Rare Pediatric Disease designation is given to drugs or biologics intended to rare diseases that primarily affect people under the age of 18.
If ATA230 is approved for congenital CMV, the company will be eligible to receive a rare pediatric disease priority review voucher, which can be used by the company to submit a drug for FDA approval and give it a priority review. In other words, that drug would be reviewed within 6 months instead of the standard 10 months.
The voucher can also be sold to other companies and that shortened 4 month review period is of great value on the free market. Earlier this year, Sarepta sold its voucher for $125 million.
Image courtesy of the CDC
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