Nanotechnology Now

Our NanoNews Digest Sponsors
Heifer International

Wikipedia Affiliate Button

Home > Press > Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-APOC3

Abstract:
Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ARO-APOC3 for the treatment of familial chylomicronemia syndrome (FCS). There is no currently available therapy in the U.S. that can adequately treat FCS.

Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-APOC3

Pasadena. CA | Posted on June 21st, 2019

ARO-APOC3 is a subcutaneously administered RNAi therapeutic targeting apolipoprotein C-III (APOC3) currently being developed as a potential treatment for patients with severe hypertriglyceridemia and FCS. The company began dosing ARO-APOC3 in a first-in-human study in March 2019. The trial is a Phase 1 single and multiple dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of ARO-APOC3 in up to 63 adult healthy volunteers with elevated triglycerides and patients with severe hypertriglyceridemia and FCS.

The mission of the FDA’s Office of Orphan Products Development (OOPD) is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. In fulfilling that task, the OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives may include a partial tax credit for certain clinical trial expenditures, the waiver of certain FDA user fees, and potential eligibility for seven years of orphan drug marketing exclusivity, if approved.

####

About Arrowhead Pharmaceuticals, Inc.
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on Twitter @ArrowheadPharma. To be added to the Company's email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts .

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. These statements are based upon our current expectations and speak only as of the date hereof. Our actual results may differ materially and adversely from those expressed in any forward-looking statements as a result of various factors and uncertainties, including the safety and efficacy of our product candidates, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, our ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in our markets, and the enforcement of our intellectual property rights. Our most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q discuss some of the important risk factors that may affect our business, results of operations and financial condition. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

For more information, please click here

Contacts:
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA
626-304-3400


Investors and Media:
LifeSci Advisors, LLC
Brian Ritchie
212-915-2578

www.lifesciadvisors.com

Copyright © Arrowhead Pharmaceuticals, Inc.

If you have a comment, please Contact us.

Issuers of news releases, not 7th Wave, Inc. or Nanotechnology Now, are solely responsible for the accuracy of the content.

Bookmark:
Delicious Digg Newsvine Google Yahoo Reddit Magnoliacom Furl Facebook

Related News Press

News and information

Nanometrics to Announce Second Quarter Financial Results on July 30, 2019 July 17th, 2019

Breakthrough material could lead to cheaper, more widespread solar panels and electronics July 16th, 2019

Caught in the act: Images capture molecular motions in real time July 15th, 2019

NUS ‘smart’ textiles boost connectivity between wearable sensors by 1,000 times: Metamaterials are incorporated into conventional clothing to dramatically improve signal strength between electronic devices, allowing for new applications July 15th, 2019

Govt.-Legislation/Regulation/Funding/Policy

Caught in the act: Images capture molecular motions in real time July 15th, 2019

An 'EpiPen' for spinal cord injuries July 12th, 2019

The best of both worlds: how to solve real problems on modern quantum computers July 12th, 2019

What happens when you explode a chemical bond? Attosecond laser technique yields movies of chemical bond dissociation July 12th, 2019

Nanomedicine

An 'EpiPen' for spinal cord injuries July 12th, 2019

Nanotechnology delivers hepatitis B vaccine: X-ray imaging shows that nanostructured silica acts as a protective vehicle to deliver intact antigen to the intestine so that it can trigger an immune response. The material can give rise to a polyvaccine against six diseases July 12th, 2019

Sheaths drive powerful new artificial muscles July 11th, 2019

Nanotechnology pioneer Chad Mirkin wins Kabiller Prize in Nanoscience and Nanomedicine: Molly Stevens of Imperial College London receives Kabiller Young Investigator Award July 11th, 2019

Announcements

Nanometrics to Announce Second Quarter Financial Results on July 30, 2019 July 17th, 2019

Breakthrough material could lead to cheaper, more widespread solar panels and electronics July 16th, 2019

Caught in the act: Images capture molecular motions in real time July 15th, 2019

NUS ‘smart’ textiles boost connectivity between wearable sensors by 1,000 times: Metamaterials are incorporated into conventional clothing to dramatically improve signal strength between electronic devices, allowing for new applications July 15th, 2019

Nanobiotechnology

An 'EpiPen' for spinal cord injuries July 12th, 2019

Nanotechnology delivers hepatitis B vaccine: X-ray imaging shows that nanostructured silica acts as a protective vehicle to deliver intact antigen to the intestine so that it can trigger an immune response. The material can give rise to a polyvaccine against six diseases July 12th, 2019

Nanotechnology pioneer Chad Mirkin wins Kabiller Prize in Nanoscience and Nanomedicine: Molly Stevens of Imperial College London receives Kabiller Young Investigator Award July 11th, 2019

Imprinted spheres fight breast cancer: Inhibition of HER2 on tumor cells by molecularly imprinted nanoparticles July 9th, 2019

NanoNews-Digest
The latest news from around the world, FREE



  Premium Products
NanoNews-Custom
Only the news you want to read!
 Learn More
NanoStrategies
Full-service, expert consulting
 Learn More











ASP
Nanotechnology Now Featured Books




NNN

The Hunger Project