- The Food and Drug Administration on Monday approved Pfizer's drug Vyndaqel as well as a separate dosage form called Vyndamax for cardiomyopathy tied to the rare disease transthyretin-mediated amyloidosis. The decision deadline for Vyndaqel was in July and Vyndamax was in November.
- Pfizer priced the therapy at a list cost of $225,000 a year, giving the pharma a chance of achieving Wall Street sales forecast of $250 million if 1,300 patients are treated and expected rebates are accounted for, Evercore ISI analyst Umer Raffat wrote in a note to clients.
- Early approval of the 61 mg single once-daily pill of Vyndamax is important for Pfizer as data suggest a 20 mg dose of Vyndaqel is also effective at delaying death and preventing cardiovascular hospitalizations. The approved dose of Vynadqel is four 20 mg pills every day.
Pfizer has been eager to talk up its pipeline in recent months as it confronts the loss of patent protection for its best-selling drug Lyrica (pregabalin).
Vyndaqel (tafamidis) has been front and center in this discussion, as it is a treatment for a rare disease that can command high prices and will face little immediate competition.
The condition it treats is often mistaken for heart disease, and Pfizer has acknowledged part of its job will be raising physician and patient awareness to increase diagnosis. The company believes as many as 100,000 people have the disease in the U.S.
In transthyretin (TTR)-mediated amyloidosis, a mutation of the transthyretin gene causes misfolding of proteins, leading to accumulation of amyloid in tissues throughout the body. One place that can occur is the heart.
Another is in peripheral nerves, causing neuropathy. Two drugs for that condition are approved by the FDA, Akcea Therapeutics and Ionis Pharmaceutical's Tegsedi (inotersen) and Alnylam Pharmaceutical's Onpattro (patisiran). Both companies are working to treat all patients with TTR-mediated amyloidosis, potentially putting the companies in competition with Pfizer over time.
Vyndaqel is the form of the drug — tafamidis meglumine — that has been sold in the EU to treat polyneuropathy since 2011 at a 20 mg once daily dose. Vyndamax, by contrast, is a free acid form of tafamidis. At a 61 mg once daily dose, it achieves the same effectiveness in cardiomyopathy as four 20 mg Vynadeqel pills.
In clinical trials, Pfizer dosed Vyndaqel at both the 20 mg and 80 mg levels. The primary analysis, which showed a statistically significant reduction in death from all causes and hospitalization from cardiovascular causes, pooled the 20 mg and 80 mg dosage groups together. Secondary analyses, however, showed that the 20 mg can produce a statistically significant benefit on hospitalizations.
Thus the 61 mg pill will be essential to ensuring there are no dose reductions, particularly among patients who experience side effects, and that the combined products achieve their commercial potential.
"We estimate that Vyndamax 61 mg capsules will be commercially available beginning in latter half of 2019, as the product is currently being manufactured," Pfizer said in a statement. "Over time, we will aim to transition all patients to Vyndamax once sufficient supply is available, given its convenient, single capsule daily dosing. The products are not substitutable on a per milligram basis."
The $225,000 price is justified by the reduction in cardiovascular complications and comparisons with other drugs, like those treating cancer, that carry similar prices, Pfizer said.
Nonetheless, given the price tag, health insurers may very well ask Pfizer to enter into value-based reimbursement agreements in which rebates would be increased if Vyndaqel and Vyndamax do not achieve some agreed-upon outcomes goals.
Pfizer CEO Albert Bourla expressed openness to such agreements during the company's first-quarter earnings call last week.