Complete Response Letters

Deleted Clinical Trial Data Derailed Applied Therapeutics’ Govorestat NDA

 

An FDA complete response letter followed a bioresearch warning letter citing the integrity of electronic data capture and handling of dosing errors in a pivotal trial of govorestat for galactosemia.

Dark Skies For Ocaliva: US FDA Sides With AdComm In Complete Response Letter

 

The US FDA rejected Intercept’s bid to convert Ocaliva’s accelerated approval in primary biliary cholangitis, meaning withdrawal prospects loom.

Vanda’s Tradipitant Gets CRL In Gastroparesis But Another NDA Filing Is Still On

 

The company will file a new drug application with the US FDA by the end of the year for tradipitant in a different indication, motion sickness.

US FDA Advisory Committee Conduct Could Take Center Stage in Lykos Appeal of MDMA Rejection

 

Sponsor of psychedelic PTSD treatment will try rarely successful formal dispute resolution process after receiving a complete response letter. 


Steady On For US FDA’s Novel Approvals At Mid-Year, But Another 2023 Is Unlikely

 

US FDA would need to approve 44 novel agents by year-end to match 2023’s big total, but only 40 candidates are known to have user fee goals in the second half of 2024.

Pharma Looks To America First: US FDA Holds Overwhelming Lead Over EMA In Novel Approvals

 

A Pink Sheet analysis finds US approval preceded European Union clearance for 80% of products approved in both areas, but when EU approval came first, it beat the FDA by a median of 13 months. US-first approvals came close to six months before the EU.

Quality Issues Star In US FDA Complete Response Letter Trio

 

Rocket Pharmaceuticals’ gene therapy Kresladi, Daiichi Sankyo/Merck’s patritumab deruxtecan, and AbbVie’s foscarbidopa/foslevodopa (ABBV-951) received CRLs because of manufacturing concerns.

PTC’s Upstaza Takes Gene Therapy Straight To Brain (And US FDA); Translarna To Return Mid-Year

 

In a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.


Vanda Appeals FDA’s Denial Of Hetlioz Jet Lag Application As It Is Hit With CRL For Insomnia

 

Vanda CEO tells the Pink Sheet that the firm is going straight to appellate court to refute agency’s rejection of jet lag indication for Hetlioz. Similar battles may lie ahead over Vanda’s applications on Hetlioz for insomnia and tradipitant for gastroparesis.

Marching On: Eight Novel Agents Among March Goal Dates For US FDA

 

Crowded calendar could bring to an end the slow start for novel approvals in 2024, as decisions come due for Lilly’s donanemab, Merck’s sotatercept, Regeneron’s odronextamab and more.

FDA’s Dry January: Little Novel Application Activity, But That Doesn’t Mean No Fun

 

US FDA’s drugs center posted one of its lowest monthly approval counts of original applications in January, but new indications filled the gap, including new claims for Merck’s Keytruda, Takeda immunoglobulins, and Sanofi/Regeneron’s Dupixent.

Astellas’ Zolbetuximab Approval Timeline Unclear After Manufacturing Issues Prompt CRL

 
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After a US FDA site inspection raised questions about the contract manufacturer of the potential first-in-class Claudin 18.2 inhibitor that could not be answered by the original January PDUFA date, Astellas will need to refile the application, but has not committed to when that might be.


Big Totals And Big Margins: US FDA Approved 72 Novel Agents In 2023 – And Acted On 90+

 

US FDA’s biologics center posted a historic 17 novel approvals, blowing past previous records, while the drug center’s 55 novel agents was close to its 21st century high.

Dry Eye Crossover Study Could Allow Aldeyra A Quick Response To US FDA’s CRL For Reproxalap

 
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Aldeyra’s bid to rely on a single Phase III trial after an initial one failed is rejected by FDA, but firm already has a special protocol assessment under review for a trial that could provide the symptom data it says agency needs to approve the RASP modulator in dry eye. 

Breakdowns In The Breakthrough Lane: When Recipients Of FDA Expedited Designation Fall Short

 

Safety signals were the most common cause of discontinued candidates in the breakthrough therapy designation program in 2023.

Data on Designations: What’s Happened To BTDs Over The Years

 

Pink Sheet infographic breaks down the breakthrough therapy designations made by the US FDA, with statistics on what’s been approved, rescinded, and everything in between.


Pink Sheet Podcast: US FDA Suffers From Shutdown Threat, Alnylam’s CR Letter, ARPA-H Wants FDA Incentives

Pink Sheet reporters and editor discuss the problems that emerged for the US FDA from just the threat of a government shutdown, take-aways on the FDA advisory committee process based on the complete response letter issued for Alnylam’s Onpattro, and incentives that the FDA could offer for ARPA-H projects that reach the application stage.

US FDA Rejects Onpattro Label Expansion: History Repeats In Reverse?

 
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In going against an advisory committee vote, the agency produces ironic echoes with the history of the approved treatment for ATTR-CM, Pfizer’s Vyndaqel.

Alnylam’s Complete Response Is Classic Example Of FDA-Advisory Committee Disagreement

 

The situation also is a perfect example of FDA doing what it says it does – paying more attention to the thinking behind advisory committee votes than the votes and vote totals themselves.

US FDA’s October Outlook: Xphozah After Dispute, Santhera’s New Steroid For DMD, & Plenty Of Pfizer

 

Fourteen user fee goal dates are on the October calendar, including three novel agents, according to the Pink Sheet US FDA Performance Tracker.