RDT receives FDA approval for new liquid form of treatment for patients with ALL

Apr 30 2014

Rare Disease Therapeutics, Inc. (RDT), the producer of orphan pharmaceuticals for the treatment of rare diseases, announced today that they have received FDA approval for PURIXAN™ (mercaptopurine) 20 mg/mL oral suspension---a new, easier-to-dose, liquid form of an established treatment for patients with Acute Lymphoblastic Leukemia (ALL). Unlike the 50 mg tablet form that healthcare professionals often split to provide children with the desired dose, the raspberry-flavored PURIXAN liquid enables the measurement of precise doses that can easily be administered to pediatric and adult patients.

On April 28, 2014, in a written announcement to Clinical Oncologists, the US Food and Drug Administration pointed out that "Compared to tablets, a suspension offers the advantage of more accurately delivering the desired dose to children with a wide range of weights using a consistent administration schedule. A suspension will allow more flexibility in adjusting the dose" and that "a commercially produced suspension is more likely to provide a more consistent dose of 6-mercaptopurine than ad hoc compounded formulations.¹"

"Our management shares FDA's opinion that a 50 mg tablet formulation is not ideal for treating the age and weight range of patients with ALL—especially children under 6 years of age," responded Milton Ellis, President & CEO of RDT. "That's why our team has been so enthusiastic about working with FDA to bring PURIXAN oral suspension to market; and why we are also grateful for the close collaboration of Nova Laboratories, Ltd., the inventor and manufacturer of the sophisticated formulation that gives PURIXAN its special clinical advantages."

Approximately 6,020 people in the United States are diagnosed with new cases of ALL each year, and the risk is highest in children under 5 years of age according to the American Cancer Society.

"ALL therapy includes 6-mercaptopurine taken orally every day for 2-3 years.  Until now, the only FDA approved formulation of this drug available in the US has been pills, which can be very hard for young children to take.  This oral-suspension formulation should be much easier for young children to take and help parents to make sure that their children get the treatment that they need to cure ALL," commented Stephen Hunger, MD, Chairman of the Children's Oncology Group ALL Disease Committee, Professor and Ergen Family Chair in Pediatric Cancer, and Director, Center for Cancer and Blood Disorders at the University of Colorado School of Medicine.

Because PURIXAN is designated an Orphan Drug, RDT has been granted a period of exclusivity in marketing this innovation and will be distributing PURIXAN oral suspension through the firm AnovoRx Distribution, LLC in Memphis, Tennessee--which can be contacted at 1-888-470-0904.

The most common adverse reaction (> 20% of patients) is myelosupression including anemia, neutropenia, and thrombocytopenia. Less common (5-20% of patients) adverse reactions include elevated transaminases, elevated bilirubin, intestinal ulceration, nausea, vomiting, anorexia, diarrhea and rashes.