In a previous article, we discussed the findings of a Linkedin poll regarding Gene Therapy challenges. Though the most common answer was manufacturing and supply chain challenges, the comments below the poll highlighted the regulatory challenges sponsors must overcome to get approval.
Indeed, bringing a Gene Therapy product to market requires navigating complex regulatory pathways, which can be time-consuming and challenging.
With Gene Therapy (GT) advancements, the regulations of EMA and the FDA around these novel treatments are also developing. Thus, discussing how sponsors can minimize regulatory challenges is of critical importance.
3 things to consider to get your Gene Therapy approved
With FDA and EMA setting specific guidelines for Gene Therapy approvals and the ongoing challenges regarding the manufacturing process, supply chain, and clinical trials, sponsors need to ensure that they:
- engage early with regulators
- stay updated with the latest guidelines
- meet CMC requirements
Sponsors should engage early with the regulators to facilitate Gene Therapy approvals.
Sponsors should interact with regulators to ensure they get early scientific advice and understand regulatory requirements regarding product development.
Sponsors can participate in the FDA INTERACT Meetings (Initial Targeted Engagement for Regulatory Advice on CBER Products) with CBER early enough (Center for Biologics Evaluation and Research) to discuss the Gene Therapy development. These meetings can provide key advice on expediting the process by saving time and money.
For example, Novartis engaged in early communication with the FDA during the development of Zolgensma, which allowed receiving expedited review status and helped speed up the approval process.
To accelerate the approval process, programs such as Parallel Scientific Advice (PSA) facilitate EMA and FDA collaboration enabling simultaneous joint discussions among the two regulatory agencies and the sponsors.
Sponsors can accelerate Gene Therapy asset development by staying updated with the latest regulatory guidance.
Sponsors should stay informed of the latest regulatory agencies’ guidance updates. For example, in November 2022, FDA’s Center for Biologics Evaluation and Research (CBER) issued a new guidance titled “Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial“.
According to this guidance, instead of separate trials, sponsors can perform one single primary umbrella trial for different versions of the same drug/ therapy (clinicalleader.com, Fda.gov). and decide which version of the drug makes it to the next phase successfully. Employing primary umbrella trials can accelerate novel therapies development.
Sponsors can prevent delays in their Gene Therapy filings by meeting CMC (Chemistry, Manufacturing, and Control) requirements.
Manufacturing and supply chain challenges was the most common answer in the LinkedIn poll we ran in the summer of 2022 to understand better Gene Therapy challenges. That is why sponsors should gather CMC data to avoid delays in Gene Therapy filings.
Around 14 FDA filings were delayed in 2020 due to partially or fully CMC-related issues. Companies such as Voyager Therapeutics, Iovance Biotherapeutics, Bluebird Bio, etc., have faced this issue as the FDA had asked for additional data on manufacturing.
To meet FDA expectations, sponsors should collaborate with licensed partners with drug development, manufacturing, and clinical supply chain expertise. Working with trusted partners ensures compliance with FDA standards and streamlines the entire process.
What does the early approval of Gene Therapies signify for the future of pharma and biotech companies?
Currently, Gene Therapy stands on a strong foundation, with a wave of recent regulatory approvals and potentially more in the near future.
Hence, sponsors should work hand-in-hand with the regulatory agencies and trusted partners to keep abreast of recent guidance.
All stakeholders’ early and coordinated engagement means safety concerns are met, and Gene Therapies are finally approved and launched in the market.
Speeding up the time it takes to bring a new Gene Therapy to the market translates into improved ROI for pharma as it stays ahead of the competition from other novel treatments targeting the same conditions. This is crucial because approval requirements are less strict for non-Gene Therapies.
In the long term, sponsors following a solid and transparent approval process without back and forths will ease HCP concerns, and the latter will be willing to adopt Gene Therapies to cure their patients. Getting early approval improves the bottom line potential for the pharma both in the short-term and long-term.
However, even if the safety profile of Gene Therapies is managed, there is a long way till they reach patients who need them due to the high costs and associated reimbursement challenges—issues that will gain attention in the years to come.
Do you want to win in the Gene Therapy space?
Or do you need a partner to help you keep up with the latest regulatory updates?
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#genetherapy #genetherapies #FDA #EMA #FDAguidance #EMAguidance #FDAApproval #EMAApproval #Biotech #drugregulatory #drugsafety #pharmamanufacturing
Sources:
Addressing the unique regulatory challenges of gene therapies
https://www.fda.gov/media/152536/download
Overcoming the Key Challenges of Gene Therapy Commercialisation – Oxford Global
FDA Releases Final Guidance On Early-Phase Cell Gene Therapy Trials (clinicalleader.com)
Regulating the gene-therapy revolution
FDA gene therapy holdups suggest closer scrutiny by agency | BioPharma Dive
Running With Scissors: The FDA Moves to Tame Gene Therapy Drug Development
Avoiding Gene Therapy CMC and Clinical Supply Issues | Fierce Biotech
FDA INTERACT Meetings Early Interactions For Cell And Gene Therapy Sponsors