- Pfizer expects the Food and Drug Administration to decide in July on approval of tafamidis in the rare disease transthyretin amyloid cardiomyopathy. Assuming the regulator gives an OK, the big pharma company will need to overcome underdiagnosis, as the condition is often mistaken for heart failure, as well as payer resistance to what is likely to be a costly drug.
- Executives said raising awareness among physicians will be key to increasing prescriptions, and seeking to diagnose patients without use of an invasive cardiac biopsy, as was used in clinical trials. Use of radionucleotide scans is one diagnostic method that can be used, they said.
- Asked during the company's first-quarter earnings call about the possibility of payers seeking outcomes-based reimbursement contracts that would force Pfizer to increase the rebate for patients who don't achieve clinical milestones, CEO Albert Bourla said the company is discussing how payers might recognize "the value this brings to patients."
Tafamidis has had a long road to approval. Pfizer bought the private group FoldRx Pharmaceuticals nine years ago to acquire the molecule, which at the time was being tested for a type of neuropathy caused by the mutation of the transthyretin gene. The genetic alteration causes misfolding of proteins, leading to amyloid accumulation in the peripheral nerves.
In 2011, tafamidis won approval in the European Union for this condition under the name Vyndaqel. The FDA, however, rejected it a year later.
As a Europe-only rare disease drug, it doesn't appear to generate enough sales to feature independently in Pfizer's financial reports. Meanwhile, two new agents, Alynlam's Onpattro (patisiran) and Akcea's Tegsedi (inotersen) have been launched in the U.S. to treat amyloid-related polyneuropathy.
Pfizer's reboot of tafamidis into a transthyretin amyloid (ATTR) cardiomyopathy treatment has been more successful, as its Phase 3 ATTR-ACT trial results were featured at the European Society of Cardiology and in the New England Journal of Medicine last year. Data from ATTR-ACT were part of Pfizer's FDA submission, which was accepted in January.
Commercially, a major challenge for Pfizer will be making sure physicians know to test patients for ATTR cardiomyopathy. The symptoms are similar to heart failure, and it is often diagnosed as such. Angela Hwang, biopharmaceuticals group president, said the key will be educating physicians about "symptomatic attributes" of ATTR cardiomyopathy that can help identify potential patients who might need additional testing.
Cardiac biopsies can be used to identify patients, but because that is an invasive procedure, Hwang said use of a radionucleotide procedure called scintigraphy to find transthyretin precursor protein in patients may be a preferred diagnostic tool.
She acknowledged that there might not be a rapid acceleration of sales: "This is going to take time to educate to bring the full benefit of tafamadis to bear."
As a rare disease drug, tafamidis is likely to carry a higher price tag than one for a more common disorder. This is a sore point with many pharmacy benefit managers, which have begun seeking "outcomes-based" reimbursement contracts that call for rebates in cases where the patients do not respond or have a limited response.
Bourla said the company has been discussing price with PBMs — and expressed openness to having arrangements based around value — but has not come to any agreements.
Heart failure drugs have been subject to value-based agreements before. Novartis in the past has agreed to discount Entresto (sacubitril/valsartan) in populations in which a goal of reducing hospitalizations is not met. And ATTR-ACT provided a target, as tafamidis reduced cardiovascular-related hospitalizations by 32% when compared to placebo.