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US FDA’s April Outlook: Decisions Pending For Rare Pediatric Diseases, New Antibiotics

 
• By Bridget Silverman

User fee goal dates in April include four novel agents with breakthrough therapy designations, including Pfizer’s hemophilia B gene therapy and Immunity Bio’s IL-15 superagonist complex for bladder cancer.

fresh-branches-of-a-bud-and-young-leaves-with-raindrops
There are not a deluge of NMEs that could be approved in April, but they offer green shoots nonetheless. • Source: Shutterstock

All of the six novel agents with April 2024 user fee goal dates at the US FDA are using an expedited review pathway, the Pink Sheet FDA Performance Tracker’s User Fee Goal Dates tracker shows: four with breakthrough therapy designations and two with qualified infectious disease product (QIDP) designations.

The breakthrough therapy designations span the therapeutic spectrum, with oncologics for bladder and brain cancers (ImmunityBio’s Anktiva and Day One Biopharmaceuticals’s tovorafenib, respectively), a treatment for a rare primary immunodeficiency (X4 Pharmaceuticals’ mavorixafor) and

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Ocaliva: Still No Clarity On Why EU Court Opposed Revocation Of Approval

 
• By Neena Brizmohun

Advanz Pharma would have had to show that the European Commission’s decision to revoke Ocaliva’s conditional marketing approval risked causing serious and irreparable harm, according to lawyers from Van Bael & Bellis.

Final Chance To Have Your Say: Take Our Reader Survey This Week

 
• By Nielsen Hobbs

This is your final call to participate in the survey to better understand our subscribers’ content and delivery needs. The deadline is 20 September.

Shape Our Content: Take The Reader Survey

 
• By Nielsen Hobbs

We are conducting a survey to better understand our subscribers’ content and delivery needs. If there are any changes you’d like to see in coverage topics, article format, or the method in which you access the Pink Sheet – or if you love it how it is – now is the time to have your voice heard.

Brazil Pilots Digital Drug Pack Inserts

 
• By Francesca Bruce

A new pilot aims to take Brazil closer to ‘digital transformation.’

More from Pink Sheet

Spain Slashes Wait Between EU Drug Authorization And Financing Decisions

 
• By Francesca Bruce

Spanish authorities say that that timelines for drug reimbursement decisions can be improved further with the right submissions from companies.

Spain Beats England To Tevimbra Reimbursement Recommendations

 
• By Francesca Bruce

Spanish authorities have published more pricing and reimbursement reports as part of a new drive to increase transparency.

Commission Taking ‘Due Account’ Of Scathing Report On EU Clinical Trial Bottlenecks

 
• By Vibha Sharma

A report by the European pharmaceutical industry criticizes the EU’s clinical trials framework, highlighting regulatory fragmentation, inefficiencies and the need for targeted reforms.