• 10 Feb 2024
• Admin
• News Article

US-FDA grants FTA to Ionis & AstraZeneca

US FDA grants fast track designation to Ionis & AstraZeneca's eplontersen for patients with transthyretin-mediated amyloid cardiomyopathy

Ionis Pharmaceuticals, Inc. announced that the US Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. The FDA grants development programmes Fast Track designation to facilitate the development and expedite the review of drugs that demonstrate the potential to treat serious conditions and fill an unmet medical need.

"Receiving Fast Track designation from the FDA reinforces our belief that eplontersen has the potential to be a transformational treatment for patients with ATTR-CM, which remains a progressive and fatal condition for hundreds of thousands of people worldwide despite available treatment options," said Eugene Schneider, M.D., executive vice president and chief clinical development officer, Ionis. "CARDIO-TTRansform is the largest, most comprehensive study ever conducted in ATTR-CM patients, with results expected as early as next year."

Sarah Walters, vice president, US cardiovascular, renal and metabolic diseases, AstraZeneca said: "We are committed to accelerating innovation and addressing the spectrum of unmet patient needs in amyloidosis. Together with our partner Ionis, AstraZeneca is building on our heritage in cardiovascular disease and RNA-targeted therapeutics to bring a potential best-in-class treatment for people living with ATTR-CM."

In December 2023, eplontersen was approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN, under the brand name Wainua (eplontersen). As part of a global development and commercialization agreement, AstraZeneca and Ionis are commercializing Wainua for the treatment of ATTRv-PN in the US and are seeking regulatory approval in Europe and other parts of the world. Wainua was granted Orphan Drug Designation in the US and in the EU for the treatment of transthyretin-mediated amyloidosis (ATTR).

The global CARDIO-TTRansform phase 3 study of eplontersen in adults with ATTR-CM is fully enrolled with more than 1,400 patients – making it the largest study in this patient population to date. The company plans to share data from the CARDIO-TTRansform study as early as 2025.

Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed and potentially fatal disease. It is caused by the accumulation of misfolded TTR protein in the cardiac muscle. Patients experience ongoing debilitating heart damage resulting in progressive heart failure, which results in death within three to five years from disease onset. ATTR-CM includes both the genetic and wild-type form of the disease. Worldwide, there are an estimated 300,000 – 500,000 patients with ATTR-CM.

Eplontersen is a LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of transthyretin, or TTR protein. The investigational medicine is currently being evaluated in the Phase 3 CARDIO-TTRansform study for hereditary or wild-type transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a progressive and fatal condition that typically leads to progressive heart failure and often death within three-to-five years from disease onset. The CARDIO-TTRansform phase 3 study is fully enrolled with more than 1,400 patients – making it the largest study in this patient population to date.

Eplontersen was recently approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN under the brand name Wainua (eplontersen).

Wainua injection for subcutaneous use 45 mg is indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has five marketed medicines and a leading pipeline in neurology, cardiology, and other areas of high patient need.