Neuroblastoma Vaccine Candidate Granted Rare Pediatric Disease Designation
A New York-based biopharmaceutical company announced that their GD2-GD3 vaccine has been granted a Rare Pediatric Disease Designation (“RPDD”) by the US Food and Drug Administration (FDA) for the treatment of neuroblastoma.
Upon approval by the FDA of the GD2-GD3 Vaccine, Y-mAbs Therapeutics, Inc. may be eligible for a Priority Review Voucher (“PRV”), said the company’s December 12, 2019, press release.
This is important news since neuroblastoma is a type of cancer that develops from immature nerve cells found in several areas of the body and most commonly affects children age 5 or younger.
The GD2-GD3 vaccine was originally developed by researchers at Memorial Sloan Kettering (“MSK”), and licensed to MabVax Therapeutics Holdings, Inc., which sublicensed the compound for the treatment of neuroblastoma to Y-mAbs in 2018.
This vaccine in combination with adjuvants can induce patients to produce their own anti-GD2 and anti-GD3 serum titers, with the purpose of preventing subsequent relapse.
The GD2-GD3 Vaccine is being tested in a single-center phase 1 clinical trial with 52 participants at MSK, where children with neuroblastoma, who are in remission, are being treated with seven subcutaneous injections during a year to prevent relapse.
Dr. Claus Moller, Chief Executive Officer noted in a press release, “We are committed to bringing the GD2-GD3 Vaccine to children with neuroblastoma, a life-threatening cancer with a clear unmet medical need.”
“We are very pleased with the designation granted by the FDA, and plan to start a multicenter Phase II trial in neuroblastoma in 2020.”
Neuroblastoma most commonly arises in and around the adrenal glands, which have similar origins to nerve cells and sit atop the kidneys, says the Mayo Clinic website.
However, neuroblastoma can also develop in other areas of the abdomen and in the chest, neck and near the spine, where groups of nerve cells exist.
Some forms of neuroblastoma go away on their own, while others may require multiple treatments. Your child's neuroblastoma treatment options will depend on several factors.
The symptoms of neuroblastoma vary depending on what part of the body is affected.
In 2012, the United States Congress effectuated a Rare Pediatric Disease Priority Review Voucher Program to incentivize pharmaceutical sponsors to develop drugs for rare pediatric diseases. A sponsor who obtains approval of a new drug application (“NDA”) or biologics license application (“BLA”) for a rare pediatric disease may be eligible for a PRV, which may be redeemed to obtain priority review for a marketing application by the owner of such PRV.
In December 2016, the House of Representatives approved the 21st Century Cures Act, which among other initiatives reauthorizes the PRV program for rare pediatric diseases until 2020.
A drug that receives a Rare Pediatric Disease Designation RPDD before October 1, 2020, continues to be electable for a voucher if the drug is approved before October 1, 2022.
Y-mAbs is a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based and immunotherapeutic products for the treatment of cancer.
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