The decision on the fate of the novel therapy for hypertrophic cardiomyopathy was delayed because the agency said it needed more time to consider the risk evaluation and mitigation strategy (REMS) for the drug.
The FDA has moved the deadline for making an approval decision about mavacamten from January to April date, the drug’s marker, Bristol Myers Squibb announced today.
The company said the FDA notified it yesterday that the PDUFA — which is pronounced pah-do-fa and comes from the initials of the Prescription Drug User Fee Amendment — was changed from January 28, 2022 to April 28, 2022.
Mavacamten has drawn a lot of attention because it is a novel therapy that addresses the cause of hypertrophic cardiomyopathy rather than just mitigate its clinical consequences, which can include arrhythmia and low blood flow out of the heart’s main pumping chamber, the left ventricle. Bristol Myers Squibb has also raised mavacamtem’s profile with a marketing campaign.
REMS are programs to reduce possible harms from a drug. The programs can range from simple information sheets to requirements for testing.
Earlier this week, the Institute of Clinical and Economic Review (ICER), the independent cost-effectiveness group in Boston, released its final evidence report on mavacamten. ICER gave the drug a “promising but inclusive” rating as a therapy in comparison to usual therapy and disopyramide.
ICER said its cost-effectiveness analysis showed that the drug should be priced at between $12,000 and $15,000. When ICER assigned a placeholder price of $75,000 and compared mavacamten to standard therapy, the incremental cost per QALY gained was $1.2 million, which is more than the standard threshold of $50,000 to $150,000 per QALY gained.