Daily Briefing

Around the nation: FDA approves first drug to treat a rare form of ALS


FDA  on Tuesday granted accelerated approval to tofersen, the first drug approved to treat a rare genetic form of amyotrophic lateral sclerosis (ALS), in today's bite-sized hospital and health industry news from California, Massachusetts, and Tennessee.

 

  • California: GoodRx on Tuesday announced that co-founders Trevor Bezdek and Doug Hirsch are stepping down as co-CEOs. According to a  Securities and Exchange Commission filing on Tuesday, Scott Wagner will serve as interim CEO while the company searches for a replacement The company announced that Bezdek will serve as chairman of the board and Hirsch will serve as its chief mission officer. In his new role, Hirsch will continue working with the company's healthcare provider and pharmaceutical clients. Bezdek will advise the company on overall strategy and work directly with its interim CEO. (Turner, Modern Healthcare, 4/25)
  • Massachusetts: FDA on Tuesday granted accelerated approval to tofersen, the first drug approved to treat a rare genetic form of ALS. Last month, FDA's  Peripheral and  Central Nervous System Drug Advisory Committee  signaled  partial support to expedite the approval of  Biogen's experimental drug. While the committee voiced concern that clinical data did not provide sufficient evidence of the drug's effectiveness, FDA staff at the time said the committee's concerns would likely not prevent expedited approval, citing "regulatory flexibility" to accelerate access to new treatments for patients with life-threatening conditions who have no alternative. The drug, which will be sold under the name Qalsody, works by targeting a mutation in a gene known as SOD1. According to FDA, less than 500 Americans are expected to be eligible for the drug at any given time. Biogen said the drug will be priced "within a range comparable to other recently launched A.L.S. treatments." For comparison, an ALS drug approved last year was priced at $158,000 annually. (George, MedPage Today, 4/25; Robbins, New York Times, 4/25)
  • Tennessee: Children who are not infected with respiratory syncytial virus (RSV) during their first year of life could have a lower chance of developing asthma later in life, according to the prospective U.S.-based INSPIRE study. Study author Tina Hartert of  Vanderbilt University Medical Center  noted that individuals who avoided RSV infection as infants were much less likely to have asthma at the five-year follow-up. "In this large, population-based birth cohort of healthy infants born at term, we showed that infants without RSV infection have a substantially reduced risk of developing childhood asthma compared with infants with RSV infection," the study authors wrote in The Lancet. "To our knowledge, INSPIRE is the first study specifically designed to test the hypothesis that not being infected with RSV during infancy decreases the risk of childhood asthma." Notably, the researchers estimated that 15% of current five-year asthma cases could be reduced by preventing RSV infections in infants. (Short, MedPage Today, 4/20)


SPONSORED BY

INTENDED AUDIENCE

AFTER YOU READ THIS

AUTHORS

TOPICS

MORE FROM TODAY'S DAILY BRIEFING

Don't miss out on the latest Advisory Board insights

Create your free account to access 2 resources each month, including the latest research and webinars.

Want access without creating an account?

   

You have 2 free members-only resources remaining this month remaining this month.

1 free members-only resources remaining this month

1 free members-only resources remaining this month

You've reached your limit of free monthly insights

Become a member to access all of Advisory Board's resources, events, and experts

Never miss out on the latest innovative health care content tailored to you.

Benefits include:

Unlimited access to research and resources
Member-only access to events and trainings
Expert-led consultation and facilitation
The latest content delivered to your inbox

You've reached your limit of free monthly insights

Become a member to access all of Advisory Board's resources, events, and experts

Never miss out on the latest innovative health care content tailored to you.

Benefits include:

Unlimited access to research and resources
Member-only access to events and trainings
Expert-led consultation and facilitation
The latest content delivered to your inbox
AB
Thank you! Your updates have been made successfully.
Oh no! There was a problem with your request.
Error in form submission. Please try again.