Once rejected by the FDA, Pfizer’s tafamidis is back and ready to disrupt a rare disease field only recently tapped by Alnylam and Ionis. And the pharma giant's entrant, now dubbed Vyndaqel, comes with a much lower price tag.
Vyndaqel and its sister formulation Vyndamax nabbed FDA nods to treat both the wild-type and hereditary forms of a rare and fatal heart disease called ATTR cardiomyopathy (ATTR-CM), Pfizer said Monday.
The oral drug will bear a list price of $225,000 per year, a Pfizer spokesman told FiercePharma. That's far lower than the $450,000 list price both Alnylam and Ionis are fielding with their rival meds Onpattro and Tegsedi, respectively. And the Pfizer meds are pills where its competitors are both injections.
Pfiizer's new duo won't go up against Onpattro and Tegsedi yet. Those two meds are currently cleared only in hereditary ATTR polyneuropathy (hATTR-PN), a neurodegenerative variant of the ATTR disease that Vyndaqel isn't approved to treat. But Onpattro is working toward approvals in ATTR-CM, while Pfizer's hoping to move Vyndaqel into the other variant, despite an FDA rejection years ago.
Pfizer considered the drug's clinical benefits and prevalence estimates, among other things, when deciding on the drug’s price, which “reflects the value these medicines provide to patients with ATTR-CM,” the company said.
The approvals came earlier than expected: Pfizer previously said it expected Vyndaqel to win a green light in July and Vyndamax in November, Cantor Fitzgerald analyst Louise Chen said in a Monday note to clients.
“With both formulations approved together and ahead of previous expectations, this bodes well and is ideal for launch and marketing of these drugs,” she said.
Credit Suisse analyst Vamil Divan agreed, saying that the early nods “should help Pfizer move out of its dark period dealing with multiple patent expirations and into a new period of growth,” he wrote in a Friday note to investors. He recently upgraded Pfizer due to “a number of underappreciated pipeline opportunities,” including tafamidis.
Pfizer has touted tafamidis as one of its “15 in 5” drugs or indications that it hopes could launch in five years and eventually cross the blockbuster threshold. Upbeat Divan estimates peak sales of $2 billion for the drug, and potentially “significantly larger than that if Pfizer is able to commercialize it successfully.” Cantor’s Chen, for her part, pegged over $1 billion in sales. Their reasons? The drugs’ winning label.
Both analysts said the physicians they talked to are impressed by tafamidis’ efficacy and safety, not to mention it’s the only drug approved by the FDA to treat ATTR-CM. In the pivotal phase 3 study, Vyndaqel significantly cut the risks of all-cause mortality and cardiovascular-related hospitalizations compared to placebo over a 30-month period. Moreover, patients on the Pfizer drug reduced the decline in performance on a walk test and in health status measured by a patient-filled questionnaire.
The drug’s final label has an indication “clearly mentioning the cardiovascular mortality and cardiovascular-related hospitalization benefit of the product,” Divan noted, and it also stated that “the frequency of adverse events in patients treated with Vyndaqel… was similar to that with placebo.”
On top of a lower sticker price, Vyndaqel safety profile also appears superior to its competitors, Chen said. “Ionis’ Tegsedi subcutaneous injection has been noted to have safety issues such as thrombocytopenia and renal function events that could encourage physicians to lean towards prescribing Vyndaquel/Vyndamax, in our view,” she said.
Vyndaqel was shot down by the FDA in ATTR-PN in 2012 on lack of data, even though it was already approved in Europe at the time and now boasts the indication in 40 countries. Pfizer isn’t giving up on adding a U.S. approval to the mix. The company “will continue to explore with the FDA a potential path forward for tafamidis in ATTR-PN,” the spokesman told FiercePharma. At the same time, Onpattro is also heading for ATTR-CM.
Even without the two rivals, though, Pfizer has its own commercial mountain to climb. The company estimates around 100,000 people in the U.S. live with ATTR-CM, but only 1% to 2% of them are properly diagnosed. The drug’s success will rely on Pfizer’s ability to increase awareness of the condition, Divan argues.
On the company’s first-quarter call, Angela Hwang, Pfizer Biopharmaceuticals’ group president, said the company’s initial focus will be on educating cardiologists and patients about the signs of the disease, while urging them to use noninvasive diagnostics such as scintigraphy instead of a heart biopsy.
Hwang acknowledged that the drug’s commercial launch will take time. But drawing a comparison to its own lung cancer drug Xalkori—which was launched when an ALK-positive diagnosis was only 1%, according to Hwang—she said the company is confident because of its “track record of success in creating new markets.”
Vyndaqel, which comes in capsules of 20 mg and dosed as 80 mg daily, will be available soon, while the Vyndamax 61-mg capsules will be launched in the second half of 2019 after supplies are built up, the Pfizer spokesman told FiercePharma. “Over time, we will aim to transition all patients to Vyndamax once sufficient supply is available, given its convenient, single capsule daily dosing,” he said.