Pfizer's new rare-disease meds reach blockbuster stage despite pandemic hurdles

 While the COVID-19 pandemic dominated headlines at Pfizer and beyond last year, behind the scenes, the company has been busy with its launch of rare heart disease meds Vyndaqel and Vyndamax. And those efforts are showing. 

Vyndaqel and Vyndamax, approved in May 2019 to treat cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM), passed the blockbuster threshold in their first full year on the market as the company has been working to raise awareness of the disease and boost diagnosis rates. In 2020, despite challenges from the pandemic, the meds generated $1.3 billion, up from $473 million the prior year. 

At the end of last year, the diagnosis rate among ATTR-CM patients was 21%, Pfizer CEO Albert Bourla said on Pfizer’s fourth-quarter earnings call on Tuesday. That compares with 2% from when the drugmaker launched its medicines, which carry list prices of $225,000 per year. 

At the end of 2020, more than 20,000 patients had been diagnosed, and 8,500 had received Pfizer's drugs, Bourla said, including some who received them at no cost. After COVID-19 shutdowns affected patient-doctor visits last year, Pfizer has seen an improvement in diagnosis rates since the third quarter, Bourla said—though the resurgence of COVID-19 cases might affect that recovery, he cautioned.

Aside from the rare-disease meds, several of Pfizer’s other growth products “contributed significantly” to the company’s performance last year, Bourla said. Revenues for blood thinner Eliquis jumped 17% to $4.95 billion, while biosimilar revenues jumped 68% to $1.53 billion. Cancer therapies Xtandi and Inlyta and anti-inflammatory agent Xeljanz all chipped in to growth, too. In all, Pfizer's sales swelled 8% last year to $41.9 billion.

For its part, Prevnar 13—the company’s pneumoccocal vaccine and best-selling product—benefitted outside of the U.S. as the pandemic drove vaccine awareness. The shot’s sales grew 11% outside the U.S. 

Meanwhile, thanks to Pfizer's move last year to spin off its Upjohn business—which it combined with Mylan to form standalone company Viatris—the pharma giant's “decade-long conversion into a pure-play" science-based company is now complete, Bourla said in a statement. 

It's that effort that enabled the company’s quick success with its BioNTech-partnered COVID-19 vaccine program, which will be a major focus for Pfizer looking forward. The company expects $15 billion in sales from the shot this year, and it’s focused on producing 2 billion doses with manufacturing partners. 

https://www.fiercepharma.com/pharma/pfizer-s-vyndaqel-vyndamax-reach-blockbuster-threshold-despite-pandemic-s-challenges