Pfizer gets fast-track approval for rare heart condition
pharmafile | June 8, 2017 | News story | Sales and Marketing | Pfizer
Pfizer’s tafamidis has already been given approval in 40 countries across the globe but it has not currently broken into the US market. This could soon to be set to change as it has received a fast-track designation for the treatment of transthyretin cardiomyopathy (TTR-CM).
The rare condition is caused by the destabilisation of the protein transthyretin, leading to amyloid fibrils being deposited in the heart. The condition has a high mortality rate, with most only expected to live between three and five years after diagnosis.
Pfizer has had a difficult path to getting the drug to market in the US. It received a complete response letter from the FDA after it submitted a New Drug Application based on early data for the drug. It turned out to be far too early, as the FDA’s advisory committee ruled that the trial data on the drug did not show enough to warrant approval.
It meant a five year delay in getting the drug back on track to reach US patients with the condition, as the FDA demanded further evidence of clinical benefit to patients before it would reconsider the application. The long wait for US’s citizens with the condition or helping those with the condition is a source of frustration as the drug has been available the EU since 2011, under the brand name Vyndaqel.
“The Fast Track designation for tafamidis is an important milestone, as there are no currently approved treatments for TTR-CM in the US,” said Brenda Cooperstone, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “We look forward to working closely with the FDA to evaluate this medicine as a potential new treatment option for patients.”
The drug is also currently being tested to determine whether it would aid patients with transthyretin amyloid cardiomyopathy tafamidis, and has reached Phase 3 trials. This trial is expected to complete in the first half of 2018.
Ben Hargreaves
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