Cell & Gene Therapies

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

 

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.

Tougher Approval Standards May Follow Vinay Prasad’s Appointment To Lead US FDA’s CBER

 

Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.

Sponsors Like START Rare Disease Pilot, Will Prasad Maintain Its Momentum?

 
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Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.

Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
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The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.


Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
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Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.

Ryoncil Chronology: Three Review Cycles, Two CRLs, One Dispute Resolution Request

 
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The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.

Gene Therapy Sponsor INTERACT, Pre-IND Meeting Confusion Lingers Despite FDA Guidance

 
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PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.

US FDA’s Makary Floats Vague ‘Plausible Mechanism’ Approval Pathway For Rare Diseases

 

Ascertaining the novelty and benefits of Makary’s new drug approval idea – along with what it would take to implement it – requires more clarity on the types of studies he envisions being used to grant and confirm “plausible mechanism’ approvals.


EU Pharmacopoeia Adopts ‘Groundbreaking’ Cell Therapy Chapter, Seeks Feedback On Alternative Microbiological Tests

 

A new chapter that will be published in the European Pharmacopoeia soon is expected to provide a comprehensive framework for the production and quality control of cell-based preparations. Meanwhile, an existing chapter has been revised to facilitate the implementation of rapid microbiological testing methods.

India Signals ‘Systemic Changes’ In CGT Regulatory Approach

 

India is pressing ahead with “big shifts” in its regulatory approach for cell and gene therapies. Reduced “layers of review”, tighter internal timelines, upcoming guidelines to facilitate CGT development and reforms in the constitution of expert committees are some of the key changes.

Japan Looks To Build Domestic Capacity In New Modalities

 
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Japan's trade ministry is providing new support to build domestic capacity for new modalities including cell and gene therapies, as part of wider efforts to support the national bioventure ecosystem.

Pipeline To Product – What Is Driving Cell And Gene Therapy Progress In India?

 
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US-based Colossal Labs has claimed to bring back the dire wolf from extinction. While the science is not as dramatic in India yet, cell and gene therapies are making progress as treatments for cancer and other diseases. Pink Sheet takes a look at what is driving CGT success and growth.


Gene Therapy ‘Survival Of The Fittest’ – Why Companies Need To Understand Health Systems

 

Cell and gene therapy manufacturers must consider the practicalities of their product within the context of a health care system before it comes onto the market to be successful, experts from Novartis, AstraZeneca and England’s National Health Service say.

Device-Like System Proposed For Low-Risk Human Cell Therapies, Tissue-Based Products At US FDA

 
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A risk-based approach to human cell therapies and tissue-based products could incentivize development and prevent bad actors from taking advantage of the current FDA system.

US Approach To Cell And Gene Therapy Regulations ‘Less Strict’ Than EU

 

Experts working in the advanced therapy space say the US has less strict criteria for regulatory pathways for cell and gene therapies than the EU, particularly for products in early development.

EU HTA Regulation: Companies Can Talk To National Authorities As ‘Substitute’ For EU-Level Advice

 

Not all companies will be able to access joint scientific consultations under the EU Health Technology Assessment Regulation, but success is still possible for those that engage with national agencies early on, says EUCOPE’s Alexander Natz.


German, Dutch And Italian HTA Processes Not Supportive Of RWD, Say Companies

 

Payers and health technology assessment bodies in the Netherlands, Germany and Italy are either unwilling to use real-world data in assessments or cannot due to their existing frameworks, say representatives from Gilead Sciences and Autolus Therapeutics.

Pink Sheet Podcast: Buyouts, Departures and Other US FDA Chaos

Pink Sheet reporter and editors discuss the early retirement and buyout offers to FDA employees and significant agency departures in the Center for Biologics Evaluation and Research and chief counsel’s office as a new commissioner moved closer to confirmation.

CBER’s Marks Says US FDA’s Rare Disease Work Expected To Win Trump Administration Support

 
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Center for Biologics Evaluation and Research Director Peter Marks expects no fundamental changes in support for FDA initiatives and said the Rare Disease Innovation Hub, which promotes cross-center collaboration, is “consistent with what we're hearing in the current environment.”

CBER Losing Cell, Gene Therapy Leadership As Workforce Culling Continues

 

Among those now leaving the US FDA’s biologics center is Lola Fashoyin-Aje, who was considered a rising star at the agency when she became director of the Office of Therapeutic Products’ Office of Clinical Evaluation in 2024.