EUSA Pharma, a transatlantic specialty pharmaceutical company focused on oncology, said that the US Food and Drug Administration (FDA) has approved its orphan drug Erwinaze (asparaginase Erwinia chrysanthemi) for the treatment of acute lymphoblastic leukaemia (ALL) in patients with hypersensitivity to E. coli-derived asparaginase. Erwinaze will be available to patients throughout the United States immediately.

ALL is the most common form of childhood cancer, with approximately 2,900 patients under the age of 20 diagnosed in the USA each year. It is also one of the most curable forms of cancer, with remission rates in treated children of over 95 per cent and 75 – 85 per cent surviving at least five years without recurrence of leukaemia. Treatment involves a number of stages and drugs, and typically includes asparaginase as an essential component of current protocols. Erwinaze is indicated as an integral part of a multi-agent regimen for the treatment of ALL patients who develop hypersensitivity to current products derived from E. coli, and is therefore the first and only approved treatment option available for patients with hypersensitivity to standard-of-care treatment with pegaspargase. An estimated 15 – 20 per cent of ALL patients develop hypersensitivity to E. coli-derived asparaginase, representing approximately 450 - 600 children in the United States each year.

“Treatment with asparaginase is a vital and life-saving therapy for thousands of patients, mostly children, with ALL each year. Unfortunately, a number of these patients develop hypersensitivity to asparaginases derived from E. coli, including pegaspargase, and are unable to complete the recommended course of treatment. The approval of Erwinaze is an important advance because it is the only treatment option that can enable these patients to continue and complete their full course of therapy,” said Stephen E Sallan MD, chief of Staff, Dana-Farber Cancer Institute and Professor of Paediatrics, Harvard Medical School.

“Today’s approval of Erwinaze highlights EUSA’s ongoing commitment to the oncology field, and the treatment of patients affected by orphan diseases. We are grateful to our partners at the UK Health Protection Agency, our investigators and employees, and to the Food and Drug Administration for their dedication to this effort and for helping us make Erwinaze available to the hundreds of patients who can benefit from it each year,” said Dr Tim Corn, EUSA Pharma’s chief medical officer.

Commenting on the news, Bryan Morton, president and chief executive officer of EUSA Pharma, said, “The approval of Erwinaze marks a major milestone for EUSA, and represents the culmination of many years of work. Launching Erwinaze to sit alongside our existing portfolio of specialty products is also a major strategic milestone for the company, as this is the first treatment EUSA has developed internally. This achievement represents a transformation for the company, signalling our transition into a specialty development as well as commercialization organization.”

The Erwinaze approval is based on the results of clinical studies in 630 ALL patients. In the pivotal efficacy study conducted in 58 subjects, 100 per cent of evaluable patients achieved the asparaginase activity primary endpoint.

EUSA will offer a patient assistance programme to help expand access to Erwinaze for patients who lack health insurance or meet certain other criteria.

Erwinaze is an asparaginase enzyme that depletes the level of asparagine in the bloodstream. Asparagine is essential for cell growth, and its removal from the blood inhibits the growth of cells associated with ALL. Asparaginase products are derived from bacteria, and approximately 15 – 20 per cent of patients develop hypersensitivity to modern products derived from Escherichia coli, preventing their continued treatment. Erwinaze, which is produced by Erwinia chrysanthemi, is immunologically distinct from these therapies and is suitable for patients with hypersensitivity to E. coli-derived treatments. Erwinaze was originally discovered by the UK Health Protection Agency, and the assays for the US Biologics License Application were conducted by AIBioTech, Richmond, Virginia.