Sigma-Tau Pharmaceuticals, Inc. has announced that it has received approval from the US Food and Drug Administration (FDA) to manufacture L-asparaginase, the primary ingredient in the oncology medicine Oncaspar (pegaspargase). Oncaspar is the only FDA-approved PEGylated formulation of L-asparaginase which is a key component in the treatment of acute lymphoblastic leukaemia (ALL).

The attempt to secure approval to manufacture L-asparaginase came after the previous supplier decided to cease production, leading to a three year development effort to create a comparable active ingredient. The approval averts a potentially dangerous drug shortage situation in the US which could have affected thousands of patients with ALL.

“To get a complex biologic medicine such as this exactly right takes a great deal of work for both the manufacturer and the FDA,” said Gregg Lapointe, chief executive officer, Sigma-Tau Pharmaceuticals, Inc. “We are pleased to announce that with this approval, there will be no interruption in either the production of the medicine or in the treatment of patients with ALL.”

L-asparaginase is an enzyme that depletes the amino acid asparagine, which certain leukaemic cells are dependent upon for survival. L-asparaginase was first approved in 1978 as a treatment for ALL. In 1994, the FDA approved the PEGylated formulation of the medicine (Oncaspar) which has the unique therapeutic advantages of sustained duration and prolonged effect over the native L-asparaginase, resulting in enhanced convenience for patients and providers. Oncaspar is given to patients with ALL as part of a multi-agent chemotherapeutic treatment regimen.

Administering L-asparaginase results in the depletion of asparagine circulating in the blood, which starves the leukaemic cells and results in their death. Oncaspar was updated to first-line indication in 2006.

Acute lymphoblastic leukaemia (ALL) is a cancer of the white lymphoid blood cells, which when normal, fight infections. It causes the body to produce abnormal and immature white blood cells that cannot accomplish this purpose. ALL is the most common form of leukaemia found in children, representing 23% of cancer diagnoses in children under age 15. It occurs in one of every 29,000 children in the United States each year. While ALL is most common in children, people can be diagnosed with ALL at any age and the risk increases after age 45. Over the past 35 years, the overall five year survival rate for children with ALL has greatly improved, going from less than 5% in the 1960s to about 85% today.

Oncaspar (pegaspargase) is the only FDA-approved PEGylated formulation of L-asparaginase, the enzyme that depletes the amino acid asparagine. For the last 25 years, L-asparaginase has been an important component in the treatment of acute lymphoblastic leukaemia (ALL). While normal cells can produce asparagine, leukaemic cells are unable to produce enough asparagine to survive on their own. L-asparaginase is given to ALL patients to ensure depletion of asparagine that is circulating in the blood. Depletion (starving the leukaemic cells) of asparagine ultimately results in leukaemic cell death.

Sigma-Tau Pharmaceuticals, Inc. is a US based, wholly owned subsidiary of the sigma-tau Group, and is dedicated solely to the global development and commercialization of medicines for patients with rare diseases. Sigma-Tau Pharmaceuticals, Inc. is based in Gaithersburg, Maryland.