The FDA wants more time to deliberate on Novartis' ($NVS) panobinostat, a multiple myeloma treatment that failed to impress a panel of independent experts, signaling a delay that could nearly negate the agency's previous promise of a speedy review.
The agency has extended its review period by as much as three additional months, Novartis said, pushing its decision deadline back from December to as late as March. In the spring, when the FDA first accepted the company's application, the agency granted panobinostat priority review status, promising to approve or deny the drug in 8 months instead of the usual 12. With the new delay, the FDA is threatening to all but wave off that designation in what amounts to the latest setback for the drug formerly known as LBH589.
Earlier this month, a panel of FDA advisers voted 5-2 against recommending the panobinostat's approval, acknowledging the drug's benefits for patients with previously treated multiple myeloma but concluding that its side effects were too severe to warrant approval. Agency reviewers are not beholden to the opinions of their independent panels, but they tend to side with the experts, putting Novartis' hopes for an eventual approval in jeopardy.
|Novartis oncology chief Alessandro Riva|
"We are committed to working with the FDA as they continue to review the LBH589 NDA," Novartis Oncology chief Alessandro Riva said in a statement. "Multiple myeloma remains an incurable cancer where patients who have relapsed or become resistant to available therapies need new treatment options."
In its Phase III trial, a combination of the company's panobinostat, Takeda's Velcade and the steroid dexamethasone prolonged progression-free survival (PFS) by 3.9 months compared to the other drugs on their own, helping patients live a median of one year without a major event. At the same time, 7% of patients in the panobinostat arm died from non-cancer complications compared to just 3.5% in other group, reporting symptoms including myelosuppression, hemorrhage, infection, gastrointestinal toxicity and cardiac toxicity.
The drug, which Novartis hopes to market as Farydak, 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.
Panobinostat is one of 10 pipeline treatments Novartis' ambitious oncology division plans to launch by 2017, and the Swiss drugmaker is on record with expectations to develop 14 blockbusters by 2018.
- read the statement