The world's biggest cancer-drugs maker Roche (RHHBY) was dealt another setback Friday. The U.S. Food and Drug Administration rejected Roche's request for accelerated approval of its trastuzumab-DM1 (T-DM1) license application. T-DM1 is a potential breast cancer treatment.
The FDA said the T-DM1 trials did not meet the standard for accelerated approval because all available treatment choices approved for metastatic breast cancer had not been exhausted in the study population.
The Swiss company said it will continue with its ongoing Phase III trial and will submit the data from the amended study to support a global regulatory submission in mid-2012. The study compares T-DM1 to lapatinib (GlaxoSmitKline's Tykerb) in combination with capecitabine (Roche's Xeloda) in people with advanced HER2-positive breast cancer whose disease has worsened after receiving initial treatment. HER2-positive breast cancer is characterized by increased quantities of the HER2 protein on the surface of the tumor cells and affects approximately 20% to 25% of patients.
High Revenue Hopes for T-DMI
T-DM1 is what's called an armed antibody as it attaches trastuzumab (Roche's Herceptin) to the chemotherapy DM1. Trastuzumab's job is to bind to the HER2-positive cancer cells and is believed to block out-of-control signals that make the cancer grow while also calling on the body's immune system to attack the cells. Once T-DM1 is absorbed into those cancer cells, it is designed to destroy them by releasing DM1. Roche's Genentech licenses technology for T-DM1 under an agreement with ImmunoGen (IMGN).
Roche submitted the application in July. A mid-stage study showed T-DMI shrank tumors in 33% of women with advanced HER2-positive breast cancer that had worsened following treatment with an average of seven prior medicines for metastatic disease.
Back then, Roche said T-DMI could bring in annual sales of roughly $2 billion and billed the application as a major milestone, according to Reuters. The FDA's cautionary tone would be disappointing for Roche and cause investors more concerns about the company's pipeline of drugs.
Creating biosimilars (generic forms of biologic drugs) to Roche blockbusters that would lose patent protection in the next five years is difficult. T-DMI was to replace Roche's Herceptin. But this is not the company's first setback. The FDA is looking at yanking its Avastin drug for breast cancer, and Britain again refused to approve Avastin for colorectal cancer on the basis of its low cost-effectiveness.
Roche stock has lost over 20% of its value so far this year. In premarket trading, Roche stock fell 0.5%. ImmunoGen shares sank some 23%.