Rare Cancer Treatment Updates From the FDA

According to a story from The Oncology Times, the US Food and Drug Administration (FDA) has been moving forward with several treatments and potential treatments for a variety of different rare cancer indications. These decisions are a reflection of a steady development pipeline of new advances in cancer treatment as well as ongoing research that has uncovered new approaches for using currently available therapies.

Advances in Multiple Myeloma

One of these announcements includes the approval of a supplemental New Drug Application for the anticancer drug carfilzomib. This application is intended to expand the dosing options for the drug to allow for once weekly use in combination with dexamethasone as a treatment for relapse or refractory multiple myeloma. Multiple myeloma is a form of blood cancer that affects plasma cells. This new indication is the result of trial data which indicated that a stronger, once weekly dose of carfilzomib and dexamethazone was able to achieve a more effective therapeutic effect in comparison to a smaller, twice weekly dose of the two drugs. The trial did not reveal any new safety concerns with this dosing level.

Advances in Chronic Lymphocytic Leukemia and Follicular Lymphoma

The FDA also recently approved a PI3K inhibitor called duvelisib. This is a dual inhibior of PI3K-gamma and PI3K-delta that is orally available. This approval is as a treatment for chronic lymphocytic leukemia in patients that have already received two different lines of therapy. This type of cancer affects white blood cells called lymphocytes The drug also gained Accelerated Approval for relapsed/refractory follicular lymphoma after two prior lines of therapy. Follicular lymphoma affects follicle center B-cells. Duvelisib does have the potential to produce serious adverse reactions, such as skin reactions, colitis, and other infections.

Advances in BRCA Mutated Prostate Cancer

The agency also granted Breakthrough Therapy designation for rucaparib as a treatment for metastatic BRCA mutated prostate cancer that is resistant to castration. Rucaparib is part of a class of targeted therapies called PARP inhibitors, which are tailored specifically for cancers that appear as a result of BRCA mutations. This inhibitor was also recently approved for the treatment of advanced ovarian cancers that are linked to BRCA mutations.

 


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