Sanfilippo Syndrome Patients, Drug Developers At Odds With US FDA Guidance

 
• By Sue Sutter
  
Generate a summary of this article
Listen to this article
Beta

Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

Barrier
An FDA draft guidance has become a barrier to developing drugs for MPS III, advocates and sponsors say. • Source: Shutterstock

Patient advocates, sponsors and clinical experts are protesting what they view as generally unworkable recommendations from the US Food and Drug Administration on development approaches and clinical trial design for new therapeutics for Sanfilippo syndrome.

Key Takeaways

  • FDA’s 2020 draft guidance on development of treatments for MPS III recommends randomized controlled trials, which disease community representatives say are infeasible.

Sanfilippo syndrome, also known as mucopolysaccharidosis type III (MPS III), is an ultra-rare enzyme defect disease that affects young children....

Pink Sheet Podcast

Learn what's happening at the US FDA. On the go.

Derrick Gingery and the team bring you a weekly "Drug Fix".

SpotifyApple PodcastsWebsite

More from Approval Standards

PS-Pathways&Standards (Free-1): Industrial Chemicals Within Us - The Ubiquity of Industrial Products in the Human Body

 
• By Nabil

Exploring How Everyday Industrial Compounds Enter Our Systems, Their Health Implications, and the Ongoing Quest for Safer Alternatives .

Diversity Action Plans: What To Submit And When

 
• By Nielsen Hobbs

With the deadline for comments on the US FDA’s draft guidance less than a month away, the Pink Sheet offers an infographic outlining what the agency is expecting in diversity action plans and when that might be implemented.

Clinical Trial Diversity Action Plans Need Timely FDA Feedback, Groups Say

 
• By Sue Sutter

The agency also should explain the potential consequences for sponsors if enrollment goals are not met, stakeholders say in comments on the DAP draft guidance. Some commenters urged the FDA to apply waiver criteria flexibly, especially for rare diseases.

Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

 
• By Sarah Karlin-Smith

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.

More from Pathways & Standards

Anvisa To Access US FDA-Reviewed Data For More Robust Evaluations In Brazil

 
• By Francesca Bruce

Brazil has also published its recent regulatory reliance regulations in English, a move designed to increase confidence in its decisions as it presses on with aligning its national practices with global best practices.

Califf: Rare Disease Hub Will Develop Methods To Guide Approvability Determinations

 
• By Sarah Karlin-Smith

The US FDA commissioner also said the hub is looking for an executive director who will work with the CBER and CDER heads, who will serve as co-leads.

Primary Biliary Cholangitis Drug Confirmatory Trials Aim To Avoid Hurdles That Tripped Up Ocaliva

 
• By Sue Sutter

The FDA and sponsors of two new accelerated approval drugs for PBC have taken steps in study design, initiation and reporting transparency to ensure timely completion; Intercept’s COBALT trial of obeticholic acid was negatively impacted by product safety labeling changes and a high crossover rate to commercial drug.