Arrowhead Pharmaceuticals’ New Drug Application Gains Food and Drug Administration Acceptance as Company Reports First Quarter Results

Arrowhead Pharmaceuticals in Pasadena said the Food and Drug Administration has accepted its New Drug Application for plozasiran, marking a significant milestone for the rare disease treatment developer.

A company statement said the FDA set a review deadline of Nov. 18, 2025, for plozasiran, which targets familial chylomicronemia syndrome, and indicated it would not require an advisory committee meeting.

“During the recent period, Arrowhead signed and closed a potentially transformational licensing and collaboration agreement with Sarepta Therapeutics and submitted our first NDA for investigational plozasiran, which was subsequently accepted for filing by the U.S. FDA,” said Christopher Anzalone, Arrowhead President and CEO.

The Pasadena-based company strengthened its financial position through an $825 million deal with Sarepta Therapeutics, including $500 million in cash and a $325 million equity investment at a 35% premium.

Under the agreement terms, Arrowhead will receive an additional $250 million over five years, $300 million in near-term payments, royalties on commercial sales, and approximately $10 billion in potential milestone payments.

Arrowhead also reported the PALISADE study met its primary endpoint and key secondary endpoints, showing significant reductions in triglycerides, apolipoprotein C-III, and acute pancreatitis incidence among patients.

Clinical trials demonstrated plozasiran’s effectiveness, with mean reductions from baseline in triglycerides of up to 73% in patients from MUIR and 86% in patients from SHASTA-2 studies, showing favorable reductions in remnant cholesterol and non-HDL-cholesterol through 15 months in the open-label extension.

Across all clinical studies, the most frequent side effects of the 25-milligram dose were COVID-19, upper respiratory tract infection, headache, Type 2 diabetes mellitus, and abdominal pain.

The statement said Arrowhead has expanded its research pipeline, initiating clinical trials for ARO-INHBE and ARO-ALK7 targeting obesity, while ARO-CFB showed near complete inhibition of complement pathway activity.

The medication works by reducing apolipoprotein C-III production, which plays a crucial role in triglyceride metabolism and has received multiple FDA designations, including Breakthrough Therapy and Orphan Drug status.

Arrowhead expects to maintain operations through 2028 with its current funding, while pursuing additional partnership opportunities for its wholly owned drug candidates.