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Zevra’s Arimoclomol US FDA Panel Nod Includes Nonclinical Data Doubts

 
• By Sue Sutter

The pivotal trial results, along with other data, support a conclusion of efficacy for Niemann-Pick disease type C, but nonclinical data are weak and cannot serve as confirmatory evidence for a single trial, the advisory committee majority said.

Confirm
Zevra's nonclinical data cannot provide confirmatory evidence. • Source: Shutterstock

Zevra Denmark A/S’ arimoclomol demonstrated a small but clinically meaningful treatment effect for Niemann-Pick disease type C (NPC) in the drug’s lone pivotal trial, a US Food and Drug Administration advisory committee said 2 August.

Key Takeaways

  • The advisory committee majority voted that the pivotal trial results and other clinical data supported arimoclomol’s efficacy in NPC.

Clinical data beyond the randomized, placebo-controlled study also provide reassuring confirmatory evidence of arimoclomol’s efficacy and safety, a majority of...

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US FDA’s June Approval Forecast: Infection Prevention, Cancer And HAE

 
• By Bridget Silverman

Upcoming FDA user fee goal dates include novel products from a busy lung cancer pipeline, a single-dose passive immunization against RSV for infants, what could be the first oral HAE acute treatment, and a first-in-class HAE preventive antibody.

Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
• By Bridget Silverman

Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.

Maybe In May: US FDA Calls Coming On Moderna Next-Gen COVID-19 Vaccine, AbbVie’s Big ADC Bet

 
• By Bridget Silverman

The 31 May goal date for Moderna’s next-generation COVID-19 vaccine looms over the month, but the US FDA's decision lineup includes three more novel agents, including AbbVie's Teliso-V antibody-drug conjugate, as well as supplemental indications like GSK's bid for Nucala in COPD.

AMR-Targeting Drug Secures EMA PRIME Designation

 
• By Neena Brizmohun

Antabio’s next-generation, broad-spectrum antibacterial, MEM-ANT3310, has made it onto the European Medicines Agency’s priority medicines scheme. The company says its product is designed to make a significant impact on the growing problem of antimicrobial resistance.

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EU Authorization Before US? Biologics Manufacturing Is Likely To Blame

 
• By Bridget Silverman

In the infrequent cases when EU authorization precedes US FDA approvals of new products, biologics manufacturing issues are usually to blame, a Pink Sheet analysis found.

Global Pharma Guidance Tracker - April 2025

 
• By Vibha Sharma and Anabel Costa-Ferreira

Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.

Keep Talking: US FDA’s Beleaguered Biologics Center Remains Vital To Regenerative Medicine

 
• By Bridget Silverman

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