Upcoming Webinars Archived Webinars Training Vitals Host A Webinar About Get Updates Contact

PTC to Acquire Marathon’s Recently Approved Duchenne Muscular Dystrophy Drug

XTALKS VITALS NEWS

DMD

DMD is a rare genetic disorder that primarily affects young boys, and Emflaza is the first treatment to be approved for the condition, regardless of the patient’s genetic mutation, in the US.

Share this!

March 17, 2017 | by Sarah Hand, M.Sc.

Amid a drug pricing controversy, PTC Therapeutics has agreed to buy Marathon Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug, Emflaza. DMD is a rare genetic disorder that primarily affects young boys, and Emflaza is the first treatment to be approved for the condition, regardless of the patient’s genetic mutation, in the US.

While US DMD patients were previously able to import a generic version of the drug from other countries for around $1,000, Emflaza’s US Food and Drug Administration (FDA) approval means patients could now pay as much as $89,000 per year for the drug. Public criticism over the price tag for Emflaza forced Marathon to put the brakes on the drug’s launch.

“Obviously we're re-examining the current price of Emflaza ... We believe a change needs to be had, but ... it's really too premature to speculate on the price level,” commented Dr. Stuart Peltz, CEO of PTC Therapeutics in yesterday’s conference call. The biopharmaceutical company is focused on developing drugs for rare diseases and other underserved patient communities.



PTC plans to pay Marathon $75 million in cash and $65 million in stock for commercialization rights for Emflaza. The company is also awaiting regulatory approval of its own DMD drug candidate, Translarna.

“With our nearly 20-year commitment to the Duchenne community, it is deeply meaningful for us to bring this critical therapy to US patients,” said Peltz. “We believe Emflaza is a disease-modifying therapy that has been shown to slow disease progression. In keeping with PTC's mission, we are excited to work with the community to raise the standard of care for DMD patients.”

Emflaza is a steroid drug designed to reduce inflammation and the immune response in all patients with DMD. The only other approved treatment for the disorder is Sarepta Therapeutics’ Exondys 51, which is only indicated for DMD patients with a specific gene mutation.

“Our goal has always been to ensure Emflaza is studied, understood and available to any Duchenne patient who needs it, and we determined that this transaction is the best path for ensuring that will happen,” said Jeff Aronin, president and CEO, Marathon Pharmaceuticals. “Now that we have achieved FDA approval of Emflaza, the focus can turn to ensuring patients have access to this important therapy. PTC is well known by the Duchenne community and is ideally positioned to achieve this shared goal.”


Keywords: DMD, Rare Disease, Drug Acquisition


| NEXT ARTICLE | MORE NEWS | BLOGS | VIDEOS | POLLS & QUIZZES | WEBINARS |

Share this with your colleagues!

MORE NEWS
Better Meal Planning for Diabetics Using a Predictive Blood Sugar App

April 21, 2017 - A new app could allow people with type 2 diabetes to make predictions about the impact of a meal on their blood sugar levels, before they even take a bite.

Featured In: Life Science News


Bacterial Biomarkers Could Make Diagnosing Colorectal Cancer Less Invasive

April 20, 2017 - Researchers at The University of Texas Health Science Center at Houston School of Public Health have identified specific strains of gut bacteria which have been associated with colorectal cancer.

Featured In: Life Science News


Weetabix Cereal Sold to Post in £1.4 Billion Deal

April 20, 2017 - The cereal was sold by Shanghai-based Bright Food, which acquired a controlling stake in the company in 2012.

Featured In: Food News

LEAVE A COMMENT
 
  
THE XTALKS VITALS LIFE SCIENCE BLOG

What Medical Device Manufacturers Need to Know Before Developing a Biological Safety Evaluation

REGISTER FOR THESE WEBINARS

Electronic Informed Consent: 2017 Industry Survey Results


Critical CRO Oversight Metrics: How to Establish the Right Metrics and Monitor them in Real-Time


The Modernization of eCOA Technology for Clinical Trials


Developing a Biological Safety Evaluation


Copyright © 2016-2017 Honeycomb Worldwide Inc.