Rare Daily Staff

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending the approval of Pfizer’s Vyndaqel for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

ATTR amyloidosis is a rare, progressive disease characterized by the abnormal buildup of amyloid deposits composed of misfolded transthyretin protein in the body’s organs and tissues. ATTR amyloidosis can impact numerous organs and tissues in the body, including the peripheral nervous system, and organs such as the heart, kidneys, gastrointestinal tract, and eyes. ATTR-CM and ATTR-PN are two presentations of the disease. On average, patients live only two to three-and-a-half years following diagnosis.

Vyndaqel is an oral transthyretin stabilizer that slows the formation of amyloid that causes ATTR-CM. Vyndaqel was granted Orphan Drug designation for ATTR-CM in both the United States and European Union in 2012 and in Japan in 2018. Vyndaqel was approved in Japan under SAKIGAKE designation for the treatment of ATTR-CM in March 2019, in the United States in May 2019, and in the United Arab Emirates in November 2019.

In 2011, a different form of Vyndaqel was approved in the European Union for transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy (ATTR-PN) to delay peripheral neurologic impairment.

The European line extension application was based on the phase 3 ATTR-ACT study, the first and only completed global, double-blind, randomized, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM.

In the primary analysis of the study, Vyndaqel demonstrated a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period in patients with wild-type or hereditary ATTR-CM. Individual components of the primary analysis demonstrated a relative reduction in the risk of all-cause mortality (30 percent) and frequency of cardiovascular-related hospitalization (32 percent) with Vyndaqel versus placebo.

The CHMP’s opinion will now be reviewed by the EC and a final decision is expected in the coming months.

“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 Brenda Cooperstone, senior vice president and chief development officer for rare disease at Pfizer Global Product Development. “In ATTR-ACT, Vyndaqel reduced mortality and the frequency of cardiovascular-related hospitalizations in patients with wild-type or hereditary forms of the disease. If approved, Vyndaqel would represent a real breakthrough for patients.”

Photo: Brenda Cooperstone, senior vice president and chief development officer for rare disease at Pfizer Global Product Development

X