FDA Roundup: New Options for Rare Conditions

Beleodaq: Peripheral T-cell Lymphoma

The FDA has approved Beleodaq for the treatment of peripheral T-cell lymphoma, a rare form of non-Hodgkin lymphoma that the agency estimates will affect 70,800 Americans in 2014. 

Beleodaq suppresses enzymes that cause T-cells to become cancerous, and is intended for use in patients who have relapsed or had unsuccessful previous treatment.

In a clinical study of 129 participants with relapsed or refractory peripheral T-cell lymphoma, 25.8% experienced complete response (the disappearance of cancer entirely) or partial response (the shrinking of tumors). 

Common side effects of Beleodaq include nausea, fatigue, fever, vomiting, and anemia. 

Reference:

FDA. FDA approves Beleodaq to treat rare, aggressive form of non-Hodgkin lymphoma [press release]. July 3, 2014. www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm403929.htm. Accessed July 8, 2014.
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NovoSeven RT: Glanzmann’s Thrombasthenia

The FDA has approved NovoSeven RT (coagulation factor VIIa [recombinant]) for the treatment of bleeding episodes in patients with Glanzmann’s thrombasthenia (GT) with refractoriness to platelet transfusions.

Glanzmann’s thrombasthenia is an extremely rare abnormality of the platelets that significantly prolongs bleeding time and has limited treatment options.

NovoSeven RT, administered as 90 mcg/kg, is intended to treat severe bleeding events and prevent bleeding during surgical procedures in patients with GT.

The efficacy of NovoSeven RT was testing using data from the Glanzmann’s Thrombasthenia Registry and the Hemostasis & Thrombosis Research Society Registry.

Overall, 92 patients were treated for 266 severe bleeding episodes and 77 were treated during 160 surgical procedures. In 94.4% of bleeding episodes and 99.4% of surgeries, NovoSeven RT was successful in preventing or stopping excessive bleeding.

NovoSeven RT carries a warning of possible serious blood clots in those using the treatment.

Reference:

Novo Nordisk. FDA approves NovoSeven RT for the treatment of Glanzmann's thrombasthenia (GT) with refractoriness [press release]. July 7, 2014. www.virtualpressoffice.com/publicsiteContentFileAccess?fileContentId=1671767&fromOtherPageToDisableHistory=Y&menuName=News&sId=&sInfo=. Accessed July 8, 2014.

–Michael Potts