FDA Approves United’s Orphan Cancer Drug

The US Food and Drug Administration has approved United Therapeutics’ Unituxin (dinutuximab) as part of first-line therapy for pediatric patients with high-risk neuroblastoma, a rare type of cancer that most often occurs in young children. United Therapeutics is a biopharmaceutical company headquartered in Silver Spring, Maryland.

Unituxin is an antibody that binds to the surface of neuroblastoma cells. Unituxin is being approved for use as part of a multimodality regimen, including surgery, chemotherapy and radiation therapy for patients who achieved at least a partial response to prior first-line multiagent, multimodality therapy. Neuroblastoma is a rare cancer that forms from immature nerve cells. It usually begins in the adrenal glands but may also develop in the abdomen, chest or in nerve tissue near the spine. Neuroblastoma typically occurs in children younger than five years of age. According to the National Cancer Institute, neuroblastoma occurs in approximately one out of 100,000 children and is slightly more common in boys. There are an estimated 650 new cases of neuroblastoma diagnosed in the United States each year.

The FDA granted Unituxin priority review and orphan product designation. Priority review shortens the timeframe for review of drug applications by four months, compared to standard reviews, and is granted to drugs that, if approved, will provide a significant improvement in safety or effectiveness in the treatment of a serious condition. Orphan product designation is given to drugs intended to treat rare diseases. With this approval, the FDA also issued a rare pediatric disease priority review voucher to United Therapeutics, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review. This is the second rare pediatric disease priority review voucher granted by the FDA since inception of the rare pediatric disease review voucher program, which is designed to encourage development of new therapies for prevention and treatment of certain rare pediatric diseases.

With its approval for Unituxin, United Therapeutics was also award a a Rare Pediatric Priority Review Voucher (PPRV) for neuroblastoma, a rare pediatric disease. The voucher can be sold (without limitation), and the holder of the voucher can redeem it with a subsequently filed new drug application or biologics license application, requiring the FDA to meet the review goals for a priority review, as opposed to a standard review. The PPRV was created in 2012 by the Food and Drug Administration Safety and Innovation Act (FDASIA) to encourage development of treatments for rare pediatric diseases. To qualify to receive a PPRV, a sponsor must submit an application for a drug or biologic intended to prevent or treat a rare pediatric disease. The rare pediatric disease application also must be eligible for priority review and rely on clinical data derived from studies examining a pediatric population and dosages of the drug intended for that population.

Source: FDA and United Therapeutics

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