U.S. FDA Accepts Shire's Biologics License Application for Calaspargase Pegol (Cal-PEG) for Acute Lymphoblastic Leukemia (ALL)

  • If approved, Cal-PEG could provide a new PEGylated asparaginase first-line treatment for patients with ALL as well as an extended shelf life helping ensure availability to patients
  • ALL accounts for approximately 75 percent of childhood leukemia in the U.S. 1

CAMBRIDGE, MA, USA I February 28, 2018 I Shire plc (LSE: SHP, NASDAQ: SHPG), the global biotechnology leader in rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for Calaspargase Pegol (Cal-PEG; SHP663). The investigational-stage compound is being reviewed
as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of December 22, 2018 for Cal-PEG.

Shire is developing SHP663 based on more than a decade of data, research and experience with ONCASPAR® (pegaspargase), which is approved in the United States as a first-line treatment for patients with ALL.2 The mechanism of action of ONCASPAR is thought to be based on selective killing of leukemic cells due to the depletion of plasma asparagine, an amino acid that certain tumor cells depend on for growth and development.2 Asparagine depletion remains a cornerstone of ALL treatment regimens. Cal-PEG is also thought to be based on plasma L-asparagine depletion. The totality of the clinical trial data submitted to the FDA for review, as part of the BLA, included a comparable safety profile and efficacy outcomes to ONCASPAR. If approved, Cal-PEG could provide a treatment that has an extended shelf life beyond that of the current PEGylated asparaginase treatment, helping ensure availability to patients.

"Today's FDA acceptance of the Cal-PEG BLA is an important milestone as we work to help address the unmet needs for rare and underserved cancers," said Andreas Busch, Ph.D., Head of Research and Development at Shire. "Developing Cal-PEG underscores our commitment to evolving the standard of care in ALL, including taking innovative steps to improve treatment options for patients."  

The BLA filing is supported by data obtained in ALL patients treated with calaspargase pegol first-line as a component of a multi-agent chemotherapeutic regimen.

Acute Lymphoblastic Leukemia (ALL)
ALL is a cancer of the white blood cells and is characterized by an excess of lymphoblasts, an immature white blood cell. Lymphoblasts are normally found in the bone marrow but can be found in the blood and other locations in people with ALL. ALL accounts for about 75 percent of childhood leukemia in the U.S. and around 78 percent in Europe;1,3 however, it can be a curable disease.4

About Calaspargase Pegol
Calaspargase Pegol (Cal-PEG) is under review with the U.S. Food and Drug Administration (FDA) as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL).

About ONCASPAR
In the United States, ONCASPAR (pegaspargase) is indicated as a component of a multi-agent chemotherapeutic regimen for first-line treatment of patients with acute lymphoblastic leukemia (ALL) and for the treatment of patients with ALL and hypersensitivity to native forms of L-asparaginase.2

Select Important Safety Information2
ONCASPAR is contraindicated in patients with a history of: serious allergic reactions to ONCASPAR, history of the following with prior L-asparaginase therapy: serious thrombosis, pancreatitis or serious hemorrhagic events.

ONCASPAR Warning & Precautions include:

  • Anaphylaxis and Serious Allergic Reactions - observe patients for 1 hour after administration; discontinue if serious allergic reactions occur
  • Thrombosis - discontinue if serious thrombotic events occur
  • Pancreatitis - evaluate for pancreatitis in patients with abdominal pain; discontinue in patients with pancreatitis
  • Glucose Intolerance - monitor serum glucose
  • Coagulopathy and Hepatotoxicity - perform appropriate monitoring

The most common adverse reactions with ONCASPAR (greater than or equal to 2%) are allergic reactions (including anaphylaxis), hyperglycemia, pancreatitis, central nervous system (CNS) thrombosis, coagulopathy, hyperbilirubinemia and elevated transaminases. Hyperlipidemia (hypercholesterolemia and hypertriglyceridemia) has been reported in patients exposed to ONCASPAR.

For additional safety information, click for the full Prescribing Information:
http://www.shirecontent.com/PI/PDFs/ONCASPAR_USA_ENG.pdf.

About Shire
Shire is the global leader in serving patients with rare diseases. We strive to develop best-in-class therapies across a core of rare disease areas including hematology, immunology, genetic diseases, neuroscience, and internal medicine with growing therapeutic areas in ophthalmics and oncology. Our diversified capabilities enable us to reach patients in more than 100 countries who are struggling to live their lives to the fullest.

We feel a strong sense of urgency to address unmet medical needs and work tirelessly to improve people's lives with medicines that have a meaningful impact on patients and all who support them on their journey.

References:

1 American Cancer Society. About Childhood Leukemia. Available at: https://www.cancer.org/cancer/leukemia-in-children/about/key-statistics.html.
2 ONCASPAR [package insert]. Cambridge, MA. Baxalta US, Inc.: 2017 Available at: http://www.shirecontent.com/PI/PDFs/ONCASPAR_USA_ENG.pdf.
3 Cancer Research UK. Children's Leukaemia Incidence. Available at: http://www.cancerresearchuk.org/health-professional/cancer-statistics/childrens-cancers/incidence#heading-Four.
4 American Cancer Society. Survival Rates for Childhood Leukemias. Available at: https://www.cancer.org/cancer/leukemia-in-children/detection-diagnosis-staging/survival-rates.html.

SOURCE: Shire

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