Pfizer has announced today that its rare disease oral drug Vyndaqel (tafamidis) has gained a new European Medicines Agency (EMA) approval for expanded use in treating adults with wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
Vyndaqel will be the first approved drug to treat this rare disease in Europe. The drug was approved by the FDA in May 2019 for the same new indication of ATTR-CM, and it was previously approved in 2011 at a lower dose for the neurological version of the disease, polyneuropathy associated with ATTR (ATTR-PN). Therefore, this new approval will dramatically increase the patient pool eligible for the drug, especially given its outstanding performance in trials. As such, GlobalData forecasts Vyndaqel to generate global sales of $3.8B by 2025 and to achieve its long-anticipated blockbuster status by 2021 with global sales of $1.5B.
The drug has presented significant results from its Phase III ATTR-ACT study, which shows that when given to ATTR-CM patients over a 30-month duration, Vyndaqel reduced the risk of mortality by 30% compared to placebo and reduced the frequency of cardiovascular-related hospitalization by 32%.
In patients with ATTR-CM, a blood protein called transthyretin is defective and breaks easily. The broken protein forms a fibrous substance called amyloid that is deposited in many organs around the body. In the walls of the heart, amyloid deposits interfere with the heart’s normal functions, causing cardiomyopathy and ultimately heart failure.
Vyndaqel is a transthyretin stabilizer that attaches to transthyretin, which prevents the protein from breaking up, thereby stopping the formation of amyloid and slowing down the progression of the disease. The drug acts on the nerves by the same mechanism of action, but its efficacy in delaying the neurological symptoms of ATTR-PN was not significant, leading to a limited sales performance prior to its new approval in cardiomyopathy.
The drug dose approved for ATTR-CM is four times higher than its dose for ATTR-PN. There are currently no alternative therapies for this rare and fatal condition besides symptomatic management. Thus, the uptake of Vyndaqel in Europe is expected to surge upon its launch, especially given its transformative results in the cardiac type of ATTR disease, its relatively low side effects, and the huge unmet need in this market.