Nova Laboratories announces FDA filing of New Drug Application for the treatment of childhood acute lymphoblastic leukemia

UK pharmaceutical manufacturer Nova Laboratories announced that the US Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for childhood acute lymphoblastic leukemia (ALL) medicine that is marketed in the EU as Xaluprine™.

The product – a liquid formulation of mercaptopurine – has been granted orphan drug designation by the FDA, a status given to a product intended for the treatment of a rare disease or condition. Nova is seeking FDA approval for the treatment of children with ALL.

Nova has partnered with Rare Disease Therapeutics, Inc. who will act as distributor for the product in North America. Nova says it hopes Xaluprine™ will be available in the US market by the second quarter of 2014.

Dr Hussain Mulla, head of clinical development for Nova, said the product had been well received by European hematologists, pharmacists and oncology nurses:

“Healthcare professionals have acknowledged that the product allows considerably more accurate doses to be administered than when using tablets and provides greater palatability than existing tablet treatments.

“The product also eliminates safety concerns around clinicians or caregivers being exposed to cytotoxic dust when splitting tablets.

“After a successful launch in Europe, we’re now looking to develop other markets, including the US, to help make life easier for children living with this disease as well as their caregivers.”

About mercaptopurine

Mercaptopurine has been used to treat children with ALL for the past 60 years. The use of mercaptopurine is considered integral to treating children with ALL and its efficacy in the treatment of ALL has been established over many years through a number of national and international trials. Consequently, all treatment protocols used by hematologists and oncologists throughout the world for treating ALL include oral mercaptopurine.

However, throughout this period an enduring difficulty for patients, caregivers and healthcare professionals, and particularly those looking after children, has been the lack of a suitable formulation of mercaptopurine. Only a 50mg tablet formulation of mercaptopurine has ever been marketed in the US but since the dose has to be adjusted according to body surface area, it has always been extremely difficult to administer the correct dose to younger children. The need for an age appropriate mercaptopurine formulation was highlighted at a FDA Meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee ( December 15th, 2009).

Xaluprine™ offers more consistent absorption and allows doses to be accurately individualized to the child’s needs – down to 2mg dose increments, whereas standard mercaptopurine tablets are 50mg. The medicine is free from artificialcolors, artificial flavoring and has no added sugar and so is particularly child-friendly. It is also easier for patients to swallow and the natural raspberry flavor ensures good palatability.

Xaluprine™ has been available in Europe since July 2012. A dedicated website, www.xaluprine.com, was launched in January 2013 to support healthcare professionals, patients and caregivers using the product.