FDA Approves United Therapeutics’ Rare Pediatric Cancer Drug and Gives the Company a Priority Review Voucher

US health regulators approved the first drug to treat pediatric patients with high-risk neuroblastoma, a rare cancer that develops on immature nerve cells.

United Therapeutics Corporation announced that the US Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) Injection, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2) and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy. The approval also came with a rare pediatric disease priority review voucher that could be worth a substantial amount of money.

Neuroblastoma usually begins in the adrenal glands but may also develop in the abdomen, chest or in nerve tissue near the spine. The cancer, which typically affects children younger than five years old, occurs in approximately one out of 100,000 children and is slightly more common in boys, according to the National Cancer Institute (NCI). It is estimated that there are 650 new cases of neuroblastoma diagnosed in the US each year. Patients with high-risk neuroblastoma have a 40 to 50 percent chance of long term survival despite aggressive therapy.

Unituxin binds to the surface of neuroblastoma cells, and data shows that the drug has the ability to extend survival for this patient population.

The drug’s safety and efficacy were evaluated in a clinical trial of 226 patients with high-risk neuroblastoma who were randomly assigned to receive either an oral retinoid drug, RA, or Unituxin in combination with interleukin-2 and granulocyte-macrophage colony-stimulating factor, which are through to enhance the activity of Unituxin by stimulating the immune system, and RA. Three years after treatment assignment, 63 percent of patients receiving the Unituxin combination were alive and free of tumor growth or recurrence, compared to 46 percent of patients treated with RA alone. In an updated analysis of survival, 73 percent of patients who received the Unituxin combination were alive compared with 58 percent of those receiving RA alone.

“Unituxin marks the first approval for a therapy aimed specifically for the treatment of patients with high-risk neuroblastoma,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Unituxin fulfills a critical need by providing a treatment option that prolongs survival in children with high-risk neuroblastoma.”

Unituxin was granted priority review and orphan product designation by the FDA. With this approval, the agency 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 voucher handed out by the agency through its program, designed to encourage development of new therapies for prevention and treatment of certain rare pediatric diseases. United Therapeutics can either use the voucher for its own drug, or the company can sell it, without limitation, to another pharmaceutical manufacturer. Previously, Sanofi and Regeneron bought a voucher from Biomarin for $67.5 million, allowing the companies to get ahead of Amgen for its PCSK9 drug. Additionally, Gilead paid $125 million for their voucher, which the company has yet to assign to a drug program.

“We are thrilled to be the recipient of this pediatric priority review voucher, and are grateful to the Creating Hope Act for its instrumental role in making this voucher program available to encourage development of therapies, like Unituxin, for children with rare conditions and high unmet need,” said Roger Jeffs, PhD, United Therapeutics’ President and Co-CEO.

Sources: US Food and Drug Administration; United Therapeutics Corporation

Last updated: 3/10/15; 1:50pm EST



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