Pfizer’s Vyndaqel scores CHMP support for transthyretin amyloidosis in cardiomyopathy patients
pharmafile | December 16, 2019 | News story | Sales and Marketing | Pfizer, Vyndaqel, pharma
Pfizer’s once-daily 61mg oral capsule formulation of Vyndaqel (tafamidis) has secured a positive opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), it has been revealed.
The recommendation is for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM), a rare and potentially fatal condition characterised by abnormal build-up in the heart of misfolded amyloid protein as well as restrictive cardiomyopathy and progressive heart failure. Affected patients live for an average of2-3.5 years.
The ruling adds to drug’s existing authorisation in the EU as a 20mg capsule formulation for the treatment of transthyretin amyloidosis in adult patients with Stage 1 symptomatic polyneuropathy (ATTR-PN) to delay peripheral neurologic impairment, secured in 2011. Phase 3 data derived from the first and only global study into ATTR-CM, where Vyndaqel showed a “significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM”.
Additionally, the drug was shown to reduce all-cause mortality risk and frequency of cardiovascular-related hospitalisation by 30%.
“The CHMP positive opinion of Vyndaqel for ATTR-CM reflects our steadfast commitment to improving outcomes for patients living with this rare and fatal disease,” said Dr Brenda Cooperstone, Senior Vice President and Chief Development Officer, Rare Disease at Pfizer. “In ATTR-ACT, Vyndaqel reduced mortality and the frequency of cardiovascular-related hospitalisations in patients with wild-type or hereditary forms of the disease. If approved, Vyndaqel would represent a real breakthrough for patients.”
Jean-Christophe Fidalgo, President of the Amyloidosis Alliance, added: “For those living with ATTR-CM, a progressive and fatal rare disease, there are currently no available pharmacologic treatments for patients. The Amyloidosis Alliance applauds the CHMP opinion, and we hope the EC will swiftly approve Vyndaqel for ATTR-CM so patients can receive timely access to this medicine.”
Matt Fellows
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