FDA accepts BLA for calaspargase pegol a proposed treatment for ALL. - Shire plc.

Shire PLC has announced that the 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 SHP 663 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. 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. 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. 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.