FDA Approves Belinostat for Rare Lymphoma

Nick Mulcahy


July 03, 2014

The US Food and Drug Administration (FDA) today approved belinostat (Beleodaq, Spectrum Pharmaceuticals) for the treatment of relapsed or refractory peripheral T-cell lymphoma (PTCL), a rare and fast-growing type of non-Hodgkin's lymphoma.

The endorsement of belinostat, which is an orphan drug, comes through the FDA's accelerated approval program and is based on overall response rate data.

The National Cancer Institute estimates that 70,800 Americans will be diagnosed with non-Hodgkin's lymphoma in 2014, and that 18,990 will die. PTCL comprises about 10% to 15% of cases of non-Hodgkin's lymphoma in North America.

Belinostat is a pan-histone deacetylase (HDAC) inhibitor. According to the manufacturer, it is differentiated from other drugs in this class because it inhibits all 3 classes of the zinc-dependent HDAC enzymes.

"This is the third drug that has been approved since 2009 for the treatment of peripheral T-cell lymphoma," said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products, Center for Drug Evaluation and Research, FDA, in a press statement. "Today's approval expands the number of treatment options available to patients with serious and life-threatening diseases."

The FDA granted accelerated approval to pralatrexate (Folotyn, Spectrum Pharmaceuticals) in 2009 for use in patients with relapsed or refractory PTCL and to romidepsin (Istodax, Celgene) in 2011 for the treatment of PTCL in patients who have received at least 1 previous therapy.

But the various treatments for PTCL are likely to fail, according to a company official.

"While important strides have been made in treating patients with PTCL, this is a deadly disease and remains an unmet medical need, with approximately 70% of patients failing current therapies," said Rajesh C. Shrotriya, MD, chief executive officer at Spectrum Pharmaceuticals, in a 2013 press statement.

The safety and effectiveness of belinostat was evaluated in a single-group clinical trial involving 129 participants with relapsed or refractory PTCL.

All participants received belinostat until either disease progression or unacceptable toxicity. The overall response rate (complete or partial) was 25.8%.

The most common adverse effects seen in belinostat-treated patients were nausea, fatigue, pyrexia, anemia, and vomiting.

Belinostat has also been investigated as a therapy in patients with unresectable hepatocellular carcinoma and ovarian cancer.

In addition, the manufacturer notes that the product is being evaluated in combination with the standard chemotherapy doublet of carboplatin and paclitaxel in a number of cancers, including non-small-cell lung cancer.

Drugs receiving accelerated approval from the FDA are subject to confirmatory trials verifying clinical benefit.


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