Gene editing technologies such as CRISPR/Cas9 have emerged as an attractive tool not only for scientific research but also for the development of medicinal products. Gene editing-based therapies have immense potential for treating a wide range of diseases, and the first therapies are already being tested in clinical trials. The treatment indications include oncological malignancies, diseases of the hematopoietic system, and metabolic disorders. This whitepaper is focusing on the regulatory challenges faced by such therapies and how they are addressed during drug development.
Gene editing technologies as a therapeutic product class are relatively new. Therefore, it often raises the question how such products should be regulated.
Source: Infiniti Research
In 2023, the US Food and Drug Administration (FDA) made significant developments in new molecular entity and biologics by approving 71 new medicines. In the EU, gene therapy products fall under the regulatory framework of advanced therapy medicinal products (ATMPs) and a total of 25 ATMPs have received approval, as of October 2023. Considering that there are hundreds of ongoing trials using gene editing, the approval number seems incredibly low.
Stringent data requirements, Novelty and uncertainty, Precision and control, Manufacturing hurdles, and Expensive approval process makes the regulatory approval a complex landscape. Regulatory landscape surrounding gene editing presents unique challenges for companies operating in this space, but why is that and what can be done?
- Follow best practices to overcome approval hurdles
- Emulate the practices of companies that have optimized and shortened the time required for regulatory approval
The approval from action due date to IND submission of Casgevy and Lyfgenia within 5 and 10 years respectively signals a new era of speed in the often-glacial world of biotech. The sky-high development costs along with small patient populations make gene therapy products expensive; therefore, they must comply with strict quality control requirements which are imposed by regulatory agencies.
In brief, while the potential to tackle diseases is enormous for gene editing, there are also numerous hurdles that necessitate careful deliberations and continuous investigations to minimize any dangers connected with the responsible advancement and application of these revolutionary technologies.
We invite you to explore our white paper, which delves into the factors that are driving faster approvals of potentially life-changing therapies.
