NEW YORK, November 18, 2016 – Y-mAbs Therapeutics, Inc. (YmAbs), an immunotherapy company discovering and developing innovative treatments for patients with cancer, today announced that it has been granted a Rare Pediatric Disease Designation by the FDA for 124I-8H9, a radiolabeled monoclonal antibody for the treatment of Diffuse Intrinsic Pontine Glioma.
“This Rare Pediatric Disease Designation is of exceptional importance to YmAbs, who has become eligible for a Priority Review Voucher upon approval of the BLA for this rare pediatric disease. While YmAbs already has a designation for the 8H9 antibody for Relapsed Neuroblastoma with CNS leptomeningeal metastasis, this designation further increases our chances of earning a PRV. YmAbs continues to focus on maximizing its valuable portfolio of rare pediatric disease assets addressing poor prognostic cancers while utilizing government programs in place for companies committed to rare population malignancy,” said Thomas Gad, Founder, Executive Chairman and President of YmAbs.”
Dr. Claus Møller, Chief Executive Officer further notes, “We are committed to bringing 124 I- 8H9 to children diagnosed with Diffuse Intrinsic Pontine Glioma, a life-threatening cancerwith a clear unmet medical need, as no available therapy exist today. We are very pleased with the designation granted by the FDA.”
About Rare Pediatric Disease Program
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 Priority Review Voucher (“PRV”), which may be redeemed to obtain priority review for a marketing application by the owner of such PRV. A PRV is fully transferrable and can be sold to any sponsor, who in turn can redeem the PRV for priority review of a marketing application in six months, compared to the standard timeframe of approximately ten months. Since the Rare Pediatric Disease Priority Review Voucher Program was initiated, the FDA has granted seven PRVs of which five have been transferred to third parties. The first Rare Disease Priority Review Voucher was granted to BioMarin Pharmaceuticals in February 2014, who sold it to Regeneron Pharmaceuticals and Sanofi SA for $67.5 million. The most recent PRV was bought by AbbVie Ltd from United Therapeutics, Inc. for $350 million.
YmAbs is a clinical stage biopharmaceutical company focused on developing new cancer treatments through immunotherapies. In addition, YmAbs utilizes its platform technologies to create next-generation humanized, affinity matured bispecific antibodies targeting GD2 and B7H3. To further improve our bispecific antibodies, we are collaborating on the development of a novel human protein tag that dimerizes T-cell engaging bispecific antibodies, which enables higher tumor binding and results in a longer serum half-life and a significantly greater T-cell mediated killing of tumor cells. Our treatments could potentially reduce longer-term toxicities associated with current chemotherapeutics and provide the potential for curative therapy even for patients with widespread disease. YmAbs’ goal is to drive multiple product candidates in select solid tumor cancers to FDA licensure. Each candidate has the potential to treat a variety of high-risk cancers. To learn more, visit www.ymabs.com.
Y-mAbs Therapeutics, Inc.
Y-mabs Therapeutics, Inc.
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