First Ever Treatment Approved for Transthyretin Amyloid Cardiomyopathy Will Cost $225,000 per Year

The first ever drug in the United States for treating transthyretin amyloid cardiomyopathy (ATTR-CM) has finally been approved by the FDA. It’s amazing news for this patient community. But, you may be wondering- how much will it cost?

The Drug

The drug was developed by Pfizer. The medicine is called tafamidis, and it was approved in two different formulations- VYNDAQEL and VYNDAMAX. VYNDAQEL has a recommended dose of 4, 20mg capsules per day. VYNDAMAX was established as a more convenient option for patients, formulated into one, 61mg capsule daily. The therapy received Breakthrough Therapy Designation in May, and just this week, received its formal FDA approval.

In clinical trials, the new therapy reduced the mortality rate of ATTR-CM patients by 30% in comparison to placebo. In addition, the frequency of AEs in the placebo and treatment groups were comparable.

The new drug, which you can read more about here, could be life-changing for patients.

The Cost

Pfizer has set a 225,000 dollar price tag for the medication per year. It’s expected that the company will make over one billion dollars on the drug by the year 2024. Comparatively, Japan has set their price for the drug at 750,000 dollars.

Pfizer says there were three factors they considered when setting the price. First, the medications transformative nature, second, the patient population it would serve, and third, the expectation that diagnosis of ATTR-CM will improve over time.

The estimate is that there are 100,000 people living with ATTR-CM in the United States. However, of those, only 1-2% have been diagnosed. Globally, between 2,000 and 5,000 individuals are diagnosed, but there is an estimated prevalence of 400,000 to 500,000. That makes the global rate of diagnosis a mere .5% to 1%.

Similar drugs, developed as a treatment for hereditary TTR amyloidosis, a related disease, have a cost of 450,000 dollars each year. These medications were developed by Ionis and Alnylam Pharmaceuticals.

Outside of the United States, VYNDAQEL is sold for 100,000 dollars per year as a treatment for another condition called familial amyloid polyneuropathy (FAP). The FDA rejected the drug as a treatment for this disease in 2012, however it is available as a therapy for FAP in 40 other countries.

Accessibility

Pfizer has stated that after discussions with insurance companies, they are confident in the fact that the 225,000 dollar price tag of the drug will be reimbursed. However, they do know that many ATTR-CM patients are on medicaid due to the fact that this disease most commonly affects the older population. For these individuals who are unable to afford high copays, Pfizer has stated that they will help patients obtain the medication through various patient assistance programs.

You can read more about this medication and its development here.


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