Getting a new drug to market is difficult, but the U.S. Food and Drug Administration (FDA) offers a number of ways to help the process along for promising drugs that have potential against serious diseases. One of these options is designation as a breakthrough therapy. This year a number of these therapies are targeted at fighting cancer.
The breakthrough designation helps expedite the process for drug development and review for the treatment of serious or life-threatening conditions. According to the FDA, the criteria for breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.
During the fiscal year 2015, the FDA’s Center for Drug Evaluation and Research received 43 requests for breakthrough designation. Of these, six were granted and 13 were denied. The Center for Biological Evaluation and Research granted three out of 10 requests for the same time period, and denied five.
One of the drugs recently granted this designation is EBV-CTL for the treatment of patients who develop a malignancy after receiving a bone marrow transplant. The malignancy — sometimes lymphoma and other cancers —develops as a complication from the Epstein-Barr virus (EBV), which causes infectious mononucleosis in people with a normally functioning immune system.
Memorial Sloan Kettering Cancer Center is conducting an ongoing Phase 2 clinical trial of the drug. Biopharmaceutical company Atara Biotherapeutics Inc. went public in February and entered into an agreement with MSKCC for the exclusive licensing rights to three clinical product candidates involved in cancers and serious infections, including EBV-CTL.
“The receipt of breakthrough therapy designation brings us one step closer to our ultimate goal of making EBV-CTL available to all patients with EBV-LPD, a serious and life threatening condition with limited treatment options,” Dr. Richard O’Reilly, chair of the department of pediatrics and chief of the Pediatric Bone Marrow Transplant Service at MSKCC said.
So for this year the FDA approved two cancer-fighting drugs that were designated as breakthrough therapies. One is Pfizer’s Ibrance for the treatment of advanced breast cancer in women and the other is Pharmacyclis Inc.’s Imbruvica, which was approved in January as treatment for Waldenström’s macroglobulinemia (WM), a rare form of cancer that begins in the body’s immune system. It is the first drug approved to fight this type of non-Hodgkin’s lymphoma.
