A heart drug that was originally proposed to the US Food & Drug Administration (FDA) for another use has far exceeded expectations, giving it a prime position in its market.
Vyndaqel, Pfizer's new heart drug to treat wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM), “nearly quadrupled Wall Street sales expectations in its first full quarter of sales in the U.S.,” reported Andrew Dunn of Biopharma Dive. The drug, which had $156 million in global sales in July, August and September, with $79 million coming from the US, has carved out a niche in a therapeutic market that includes therapies from Alnylam Pharmaceuticals and Ionis Pharmaceuticals.
Approved in May, Vyndaqel had been rejected by the FDA in 2012 as a treatment for familial amyloid polyneuropathy, although other countries approved it for that indication. Also approved in May was Pfizer’s Vyndamax, a variant of Vyndaqel, which the company launched in September. While the Vyndaqel dose is four 20-mg capsules per day, Vyndamax is a single pill.
Wall Street projected $21 million in US sales for Vyndaqel, but a faster-than-expected rate of diagnosis for the rare disease treated by the drug may have contributed to its popularity, said Evercore ISI analyst Umer Raffat. Although Vyndaqel sales were boosted by the launch, Pfizer's total revenues for the third quarter dropped 5 percent from 2018. Pfizer is narrowing its business focus to biopharmaceuticals, having dropped its consumer healthcare unit on July 31 and being ready to close a deal in 2020 with Mylan to shed its Upjohn legacy business. CEO Albert Bourla said that Pfizer will become "a smaller, science-based company." The biopharma business posted 7 percent year-over-year growth for the third quarter.
While Vyndaqel sales were $259 million through the first nine months of 2019, the third-quarter surge comes from "robust growth in all markets, including ongoing TTR-CM launches in U.S. and Japan and increased use of Vyndaqel in Europe, likely driven by increased use in mixed phenotype patients," according to SVB Leerink analyst Mani Foroohar. He added that Pfizer's drug has a slightly different indication than the competing therapies in Alnylam's Onpattro (patisiran) and Ionis' Tegsedi (inotersen), both approved to treat peripheral nerve damage from hereditary ATTR. All are expensive, with average annual list prices set at $225,000 for Vyndaqel and an average of $450,000 for Onpattro or Tegsedi.
According to Pfizer, transthyretin amyloid cardiomyopathy is a rare, life-threatening disease that is characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart. It is defined by restrictive cardiomyopathy and progressive heart failure. Previously, there were no medicines approved to treat ATTR-CM; the only available options included symptom management, and, in rare cases, heart (or heart and liver) transplant. It is estimated that the prevalence of ATTR-CM is approximately 100,000 people in the US. At the time of Vyndagel’s approval, Pfizer estimated that only one to two percent of patients with transthyretin amyloid cardiomyopathy were being diagnosed with the disease.