Monthly Archives: July 2014

Multiple Myeloma Compound Gets Orphan Drug Status

Dr J S Macdonald MDDr. John S. Macdonald
Senior Consultant for The Academic GI Cancer Consultant Consortium (AGICC) & The Academic Myeloma Consortium (AMyC) of The Oncology Consortia of CRITERIUM, INC. 

The US Food and Drug Administration has granted orphan drug status to an experimental compound for multiple myeloma known as KRN5500.

This is the second orphan drug designation for KRN5500, which is a novel, intravenous, nonopioid, nonnarcotic compound in phase 2 clinical development by DARA BioSciences.

Earlier this year, KRN5500 received orphan status for the treatment of chemotherapy-induced neuropathic pain refractory to conventional analgesics in patients with cancer.

“It is noteworthy in this regard that up to 20% of myeloma patients have intrinsic peripheral neuropathy, an incidence that increases to the range of 75% in patients treated with neurotoxic drugs such as thalidomide or bortezomib,” said David J. Drutz, MD, chief executive officer and chief medical officer of DARA BioSciences, in a statement.

In 2011, KRN5500 was granted a fast-track designation, a process designed to facilitate development and to expedite the review of drugs to treat serious conditions and fill an unmet medical need.FDA-Logo

The therapeutic potential of KRN5500 has been demonstrated in vitro and in vivo. The agent, which is a spicamycin derivative, exhibits antimyeloma effects by impairing both myeloma cells and osteoclasts, the company reports.

The orphan drug designation is “an important step toward the potential treatment of multiple myeloma and one of its major complications,” Dr. Drutz said. It will also help in the company’s “ongoing pursuit of partnering opportunities to assist in funding the clinical advancement and development pathway of KRN5500.”

Dr. John S. Macdonald comments on this important FDA decision…
“The fast tracking of this spicamycin derivative recognizes an agent that not only possesses anti cancer (multiple myeloma) activity likely to be beneficial in patients resistant to standard treatment, but also has the potential to decrease a very serious complication of myeloma, bone fractures because it inhibits osteoclast activity.”

SOURCE: Original article by Megan Brooks for MedScape http://www.medscape.com/viewarticle/826882

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