Going against the advice of its independent experts, the FDA approved Novartis' ($NVS) new blood cancer drug for patients with particularly deadly forms of multiple myeloma, looking past some serious safety issues and concerns about the drug's supporting data.
The FDA's nod clears panobinostat for use on patients with multiple myeloma who have already failed two lines of treatment, including Takeda's blockbuster Velcade. Marketed as Farydak, the drug is meant to be used alongside Velcade and the anti-inflammatory dexamethasone, the agency said, carrying a black-box warning tied to risks of severe and fatal cardiac side effects.
Last year, the FDA's Oncologic Drugs Advisory Committee voted 5-2 against approving panobinostat, noting its demonstrable benefits for patients with blood cancer but ultimately concluding that its side effects were too severe to warrant approval. The committee also criticized Novartis for handing in incomplete data and took issue with the company's analysis of results from its 768-patient Phase III trial. Later that month, the FDA added three months to its panobinostat review time, virtually rescinding the priority review designation granted to the drug and seeming to dim its prospects for approval.
But, in that time, Novartis came back with data on 193 multiple myeloma sufferers who had failed on Velcade and an immunomodulatory agent. In that group, patients who got panobinostat in addition to the other drugs posted progression-free survival of 10.6 months compared to 5.8 months for those taking Takeda's drug and dexamethasone alone. And 59% of patients in the panobinostat arm saw their cancer shrink or disappear during treatment, beating the other group's 41%.
And that was good enough to sway the FDA in another affirmation of oncology chief Dr. Richard Pazdur's penchant for rapidly approving cancer treatments that could improve the standard of care for some of the most dire patients.
"Farydak has a new mechanism of action that distinguishes it from prior drugs approved to treat multiple myeloma, making it a potentially attractive candidate agent for the treatment of multiple myeloma," Pazdur said in a statement. "Farydak's approval is particularly important because it has been shown to slow the progression of multiple myeloma."
The drug works by blocking both histone and non-histone deacetylase enzymes--abbreviated as HDACs and DACs--putting serious stress on cancer cells until they die, all while leaving healthy cells unmarred. Novartis believes its "pan-DAC" inhibitor stands out among similar efforts from Celgene ($CELG) and MorphoSys.
- read the FDA's statement