You are here

FDA Panel Says ‘No’ to Multiple Myeloma Compound Panobinostat

Reviewers question risk–benefit profile

The FDA’s Oncologic Drugs Advisory Committee (ODAC) did not recommend approval of the investigational compound panobinostat (LBH589, Novartis), a pan-deacetylase (pan-DAC) inhibitor, for patients with previously treated multiple myeloma when used in combination with bortezomib and dexamethasone.

The vote was 5 to 2 against the drug.

According to documents released by the FDA before the vote, the question at hand was not whether the treatment was effective, but whether its risks outweighed its benefits.

The committee’s opinion will be considered by the FDA in its review of the new drug application for panobinostat, but the agency is not bound to follow its advisors’ guidance.

Data presented to the committee included the results from two clinical studies that evaluated panobinostat in combination with bortezomib and dexamethasone in patients with relapsed or relapsed/refractory multiple myeloma: a phase III randomized, double-blind, placebo-controlled, global registration trial (PANORAMA-1 [PANobinostat ORAl in Multiple MyelomA]) and a phase II U.S. single-arm, open-label study (PANORAMA-2).

Multiple myeloma is an incurable disease in which the normal epigenetic process is disrupted, resulting in the growth of cancerous plasma cells, potential resistance to treatment, and ultimately disease progression. It typically occurs in individuals 60 years of age or older, with few cases in individuals younger than 40.

If approved, panobinostat would be a first-in-class therapy for patients with previously treated multiple myeloma. As an epigenetic regulator, the drug may help restore the dysregulated cell programming that occurs in the disease.

Because panobinostat is an investigational compound, its safety and efficacy profiles have not been established.

Sources: Novartis; November 7, 2014; and BioSpace; November 6, 2014.

Recent Headlines

Scenesse is new treatment for people with rare, painful light sensitivity disease
Humira, Rituxan top list of drugs that added $5.1 billion to nation's health care bill
Lower court rulings cleared the way for generic versions of the MS drug
Maryland man wins lawsuit that alleges that the company's antipsychotic caused his gynecomastia
Antidepressants, ADHD meds are also used to self-poison
Descovy joins Truvada, another Gilead product, in the HIV prophylaxis market
Data show PTC Therapeutics drug preserves lung function
U.S. maintains measles elimination status as NY outbreak ebbs
Study lists steps that could save close to $300 billion a year