Vyndaqel Is Safe, Effective for People with TTR-FAP, Review Study Suggests

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by Alice Melao |

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Treatment with Vyndaqel (tafamidis) is a safe, non-invasive way to delay neurologic progression of transthyretin familial amyloid polyneuropathy (TTR-FAP), and preserves patients’ nutritional status and quality of life, a review study shows.

The study, “Tafamidis, a Noninvasive Therapy for Delaying Transthyretin Familial Amyloid Polyneuropathy: Systematic Review and Meta-Analysis,” was published in the Journal of Clinical Neurology.

TTR-FAP is a rare, inherited disorder caused by genetic mutation in the TTR gene and consequent accumulation of TTR protein amyloid clumps in nerve cells.

This disease is marked by progressive degeneration of sensory and motor nerve cells, leading to pain and impaired sensation in the lower extremities, motor impairment, muscle weakness, and progressive dysfunction of the gastrointestinal, urogenital, and cardiovascular systems.

Vyndaqel is a therapy developed by Pfizer that can stabilize the TTR protein, preventing it from forming toxic aggregates and deposits in tissues.

It is currently approved in more than 40 countries for the treatment of FAP. In the United States, the treatment is still under development and is being evaluated for treatment of  TTR-related heart disease as well.

This oral treatment has been shown to halt protein accumulation in the heart and peripheral nerve cells, and prevent disease progression with similar effectiveness as a standard liver transplant.

Chinese researchers reviewed clinical data collected from six randomized, controlled trials that evaluated the safety and efficacy of 20 mg of Vyndaqel self-administered once daily in comparison to a placebo.

“The present study is the first systematic literature review and meta-analysis performed (…) with the aim of improving the medical evidence base for applying [Vyndaqel] as a treatment option for TTP-FAP,” researchers stated.

The pooled clinical data showed that treatment with Vyndaqel could prevent progressive loss of lower limb function compared to placebo, with a mean difference of 3.01 points in the Neuropathy Impairment Score-Lower Limbs (NIS-LL).

A positive result was also reported regarding patients’ quality of life, as determined by a significant difference of 6.67 points in the Norfolk Quality of Life-Diabetic Neuropathy total quality of life (TQOL) score between Vyndaqel and placebo groups.

Patients’ wasting status and gastrointestinal function were also protected with Vyndaqel treatment, as demonstrated by the positive difference in the modified body mass index (mBMI) compared to the placebo-treated group.

“This systematic review and meta-analysis of six randomized clinical trials has demonstrated that [Vyndaqel] exhibits a slower neurologic disease progression and better preservation of nutritional status and quality of life,” researchers stated.

According to the trials’ pooled data, Vyndaqel had acceptable safety and tolerability profiles, with similar frequencies of adverse events in both patient groups. However, fatigue was reported by 10% of patients in the placebo group compared to 0.1% in Vyndaqel group.

The proportion of patients reporting hypesthesia (diminished physical sensation, mostly affecting the skin) also differed significantly between the groups, being more frequent among placebo-treated patients.

“These findings indicate that [Vyndaqel] might be a safer, noninvasive option for patients with TTR-FAP,” researchers said.