Pfizer’s rare disease drug Vyndaqel may be its least well known blockbuster medicine. The pharmaceutical company’s COVID-19 vaccine became a household name during the pandemic, while its other top-sellers for blood clots, breast cancer and pneumococcal infections are routinely and widely advertised on TV.
Yet during the first quarter, it was Vyndaqel that outperformed expectations, delivering sales that substantially exceeded Wall Street forecasts. The drug, which treats the cardiac form of a rare disease called transthyretin amyloidosis, brought in $1.1 billion between January and March, 25% higher than the consensus estimate of just over $900 million.
Worldwide sales were $686 million during the same period last year. In a Wednesday earnings statement, Pfizer said Vyndaqel’s growth was due to “continued strong uptake,” primarily in certain European countries and in the U.S., where it is sold as Vyndamax.
Salim Syed, an analyst at Mizuho Securities, wrote in a client note that uptake may have been boosted by the Inflation Reduction Act, which capped out-of-pocket costs for people covered by Medicare Part D. Syed estimated the cap taking effect in 2024 should reduce out-of-pocket spending by about $10,000.
The strong sales prompted several analysts, speaking on a conference call, to press Pfizer executives on the drug’s remaining patent life. The basic product patent is set to expire at the end of this year in the U.S., and in 2026 in Europe.
Several generic drugmakers filed last year for U.S. approval of copycat versions, and Pfizer has sued them for patent infringement.
The company also hopes to extend the drug’s market exclusivity through 2028, and has filed requests for patent term extensions that are currently pending. If granted, the extension could help protect billions of dollars in annual sales for Pfizer as the company resets following sales declines for its COVID vaccine and antiviral drug.
Those COVID sales declines continue to weigh on Pfizer, which reported total revenue in the first quarter of $14.9 billion, down about 20% from the same period a year ago. Excluding COVID products, revenue was up 11% operationally.
Vyndaqel’s sales growth, and its looming patent questions, are also important to would-be competitors BridgeBio Pharma and Alnylam Pharmaceuticals. BridgeBio is currently awaiting a Food and Drug Administration approval decision for its drug acoramidis in the same condition Vyndaqel treats. A verdict is due from the agency by late November.
Meanwhile, Alnylam is anticipating results by June or July from an important study of its drug vutrisiran, the success or failure of which will determine whether it could compete with Vyndaqel.
Shares in BridgeBio were up nearly 5% in Wednesday morning trading, while Alnylam’s rose by about 2%.