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Imatinib Mesylate Approved in the US for Five Rare Life-Threatening Disorders With Limited Treatment Options

EAST HANOVER, N.J., Oct. 19 /PRNewswire-FirstCall/ -- In another important milestone, Gleevec/Glivec(R) (imatinib mesylate) tablets has received US regulatory approval to help patients with five distinct and potentially life-threatening disorders, representing the first time that a regulatory authority has ever simultaneously approved one targeted medicine for so many disorders.

With today's decision, and in only five years, Gleevec has now been approved in the US for seven diseases, including two solid tumors and five blood disorders with molecular targets known to be inhibited by the drug.

All of the diseases covered in the new approval by the US Food and Drug Administration (FDA) are rare and potentially life threatening. For many of the patients who suffer from them, few, if any approved treatments were available prior to Gleevec.

"The effectiveness of Gleevec in these five diseases further underscores how cancers and diseases of different origin and location can share common pathways that often respond to the same targeted treatment," said Diane Young, MD, Vice President and global head of Clinical Development at Novartis Oncology. "These approvals further build and demonstrate our historical commitment to developing therapies for patients with rare diseases such as acromegaly, carcinoid syndrome and gastrointestinal stromal tumors."

The FDA approvals are based on data from Novartis-sponsored clinical studies and clinical data from independent medical researchers showing the efficacy of Gleevec in the treatment of these diseases, in which there is a suggested connection between a Gleevec-sensitive pathway and a disease.

Gleevec targets the activity of proteins called tyrosine kinases that appear to play important roles within some cancer cells. Gleevec has been shown to inhibit the function of the tyrosine kinase Bcr-Abl in patients with certain forms of blood cancer -- Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) -- and the receptor tyrosine kinase Kit in Kit-positive GIST (gastrointestinal stromal tumor).

Researchers have found Gleevec also inhibits other tyrosine kinases, including platelet-derived growth factor receptor (PDGFR), which have been shown to be activated in disease pathways that underlie a number of rare hematologic diseases, as well as some solid tumors.

The new diseases for which Gleevec received approval include one solid tumor and various rare blood disorders. The solid tumor is dermatofibrosarcoma protuberans (DFSP), a type of tumor that begins as a hard lump found in the skin of the chest, abdomen or leg. The four blood diseases include:

  • Relapsed/refractory Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), a rapidly progressive blood cancer characterized by the presence of the Philadelphia chromosome
  • Certain forms of myelodysplastic/myeloproliferative diseases (MDS/MPD), which involve certain blood cells made in the bone marrow
  • Hypereosinophilic syndrome/chronic eosinophilic leukemia (HES/CEL), which is characterized by the persistent overproduction of eosinophils, a certain type of white blood cell
  • Aggressive systemic mastocytosis (ASM), which is marked by the presence of too many mast cells, a certain type of white blood cell.
An approval for newly diagnosed patients is still under review by the FDA. In the European Union (EU), Gleevec was recently approved for treatment of certain patients with Ph+ ALL as well as for adult patients with a form of DFSP. The EU is also reviewing applications for approval of Gleevec as a treatment for the three other diseases MDS/MPD, HES/CEL and ASM.

Source: Novartis

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Decision supported by data from more than 4,000 patients