The multiple myeloma drug panobinostat has received a positive opinion from the European Medicines Agency (EMA) for the treatment of relapsed or refractory disease in patients who have received at least two treatment regimens including bortezomib and an immunomodulatory agent.
Panobinostat inhibits histone deacetylase, resulting in increased acetylation of histone proteins. If approved in the EU, the drug, which is marketed by Novartis Europharm as Farydak, will be the first in its class of anticancer agents. Panobinostat was previously designated an orphan medicine in view of the rarity of multiple myeloma.
The positive opinion is based on the PANORAMA-1 trial
, which included 768 patients with multiple myeloma who were randomly assigned to receive a combination of panobinostat, bortezomib and dexamethasone, or bortezomib and dexamethasone alone.
Patients who received panobinostat has significantly longer progression-free survival (PFS) than those who did not; overall survival data is still accruing. The most common side effects in patients receiving panobinostat were blood disorders, haemorrhage, diarrhoea, nausea, vomiting and fatigue.
Owing to the risk for severe toxicity, the EMA’s Committee for Medicinal Products for Human Use (CHMP) considered that the overall risk-benefit balance was positive only in patients with relapsed or refractory multiple myeloma who had received at least two prior regimens including bortezomib and an immunomodulatory agent.
Following the CHMP’s positive opinion, panobinostat is expected to be granted EU marketing authorisation within three months and its orphan status will be reviewed. A post-marketing plan to monitor the safety of panobinostat and lower its risks was also agreed by the CHMP.
 San-Miguel JF, Hungria VTM, Yoon S-S et al. Panobinostat plus bortezomib and dexamethasone versus placebo plus bortezomib and dexamethasone in patients with relapsed or relapsed and refractory multiple myeloma: a multicentre, randomised, double-blind phase 3 trial. The Lancet Oncology 2014. doi: http://dx.doi.org/10.1016/S1470-2045(14)70440-1.