Pfizer Ireland marks the approval of VYNDAQEL ® (tafamidis)

Pfizer Ireland marked the approval for VYNDAQEL ® (tafamidis). A once-daily 61 mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis. This is in adult patients with cardiomyopathy (ATTR-CM). They are hosting an expert panel led symposium at the VYNDAQEL ® 61MG launch meeting.

The recently held launch meeting heard from an array of speakers including Prof. Emer Joyce, Heart Function Cardiologist at the Mater Hospital; Dr. Ross Murphy, Consultant Cardiologist at St. James’s Hospital; Dr. Pablo Garcia-Pavia Director of the Inherited Cardiac Diseases and Heart Failure Unit at Hospital Universitario Puerta de Hierro; in addition to Dr. Sarah Cuddy, Cardiologist at the Brigham and Women’s Hospital Amyloidosis Program as well as Instructor of Medicine at Harvard Medical School.

VYNDAQEL ® is the first and only treatment launched in Ireland for patients with ATTR-CM.

Previously, treatment options for patients with ATTR-CM were restricted to symptom management. In rare cases, heart (or heart and liver) transplant.

Speaking after the virtual symposium, Professor Emer Joyce. Heart Function Cardiologist at the Mater Hospital, said ‘‘Cardiac amyloidosis and specifically ATTR-CA, affects a growing population of patients encountered in our clinical practice. With the advent of contemporary non-invasive imaging techniques, diagnostics in this field have substantially improved, allowing an earlier detection of affected individuals.’’

‘‘This alongside the emergence of effective specific therapies for ATTR-CA is expected to translate into improved outcomes. Therefore making possible a promising future for amyloid patients’’, Professor Joyce added. ‘‘It is hoped that with dedicated funding and National support as well as coordination, Ireland can be a future leader in ATTR amyloidosis care and best practice.’’

The symposium heard perspectives from the expert panel on diagnosis & management of ATTR-CM patients, patient case examples and results of long-term VYNDAQEL® data & follow up of ATTR-CM patients.

Nicola Meehan, Rare Disease Medical Lead, Pfizer Healthcare Ireland said; ‘‘I am proud that Pfizer are now in a position to positively contribute to the treatment of this condition, with the approval of VYNDAQEL ® in Ireland. It also gives patients some much-needed hope. With many patients never receiving a diagnosis or only receiving a diagnosis after symptom progression, Pfizer is committed to making a difference for patients with ATTR-CM by partnering with the community to ensure accurate and earlier diagnoses.”

ATTR-CM Prevalence and VYNDAQEL ® Approval

ATTR-CM is a progressive disease that can often be overlooked as a cause of heart failure. It is caused by unstable transthyretin proteins that misfold and aggregate into amyloid fibrils that build up in the heart and other parts of the body. The buildup of amyloid causes the heart muscle to stiffen over time, eventually leading to heart failure.  The exact prevalence of the disease remains difficult to evaluate. ATTR-CM is significantly underdiagnosed and a statistically insignificant number of observational cohort studies have been conducted.

Once diagnosed with ATTR-CM, if left untreated, and dependent on sub-type, patients have a median life expectancy of approximately two to 3.5 years. With VYNDAQEL®, patients whose only prior options included symptom management, and in rare cases, organ transplant, now have a breakthrough oral therapy that may help them live longer with fewer cardiovascular-related hospitalisations.

Pfizer, VYNDAQEL ® & ATTR-CM Pathology

The approval of VYNDAQEL ® in Ireland is based on results from the Phase 3 ATTR-ACT study. The first and only completed global, double-blind, randomized, placebo-controlled clinical trial. It is to investigate a pharmacologic therapy for the treatment of ATTR-CM. The study compared patients who received an oral daily dose of VYNDAQEL ® (tafamidis meglumine) 20 mg or 80 mg compared to those who received placebo. In the pivotal Phase 3 ATTR-ACT study, VYNDAQEL ® demonstrated a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospital stays. This is compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM (p=0.0006). Importantly, results also showed that VYNDAQEL ® reduced the decline in health status and functional capacity in as early as six months and continuing through 30 months.

There are two sub-types of ATTR-CM. Hereditary, which is caused by a change (or “mutation”) in the transthyretin gene. The symptoms beginning as early as 50 to 60 years old; or the wild-type form, which is associated with aging. It is thought to be more common, usually affecting men after age 60.

The Case for VYNDAQEL ® (tafamidis)

Tafamidis is included in several recommendations for the treatment of ATTR-CM. This is including the published recommendations of the Working Group on Myocardial and Pericardial Diseases of the European Society of Cardiology (ESC), the ESC Heart Failure Guidelines, the American College of Cardiology Recommendations (ACC) and by a panel of international ATTR-CM pathology experts.

VYNDAQEL ® was well tolerated in the Phase 3 ATTR-ACT study, with an observed safety profile comparable to placebo. The frequency of adverse events in patients treated with VYNDAQEL® was generally similar and comparable to placebo. The launch is also based on findings from an evaluation of the free acid form of tafamidis 61 mg. It demonstrated that one 61 mg capsule of tafamidis free acid corresponds to an 80 mg tafamidis meglumine dose (4 x 20 mg capsules). The safety of the 61 mg dose was not evaluated in ATTR-ACT. The tafamidis 61 mg capsule was developed for patient convenience to enable a single capsule for daily administration.

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Click here  or scan the QR Code to view a recording of the Expert Panel Symposium at the VYNDAQEL® 61MG Launch Meeting:

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