Orphan Drug Designation for Gene Therapy Products

Explore the intricacies of orphan drug designation and its impact on gene therapies for rare diseases. Unlock vital insights into innovative treatment solutions.

The innovation of new treatments within the life science industry is key, and gene therapy products are one bright future within the scope of treating diseases and conditions very rarely found. Innovative therapies like this can be truly life-changing, but present unique regulatory challenges. 

Among the variety of regulatory pathways available, orphan drug designation is one of the most important; this can offer a financial incentive to sponsors developing gene therapy products for the treatment of rare disease conditions. 

In this article, we will take a closer look into the complex world of orphan drug designation pertaining to gene therapy products and will discuss the insight of the regulatory considerations concerning such a status. 

Orphan drug designation is a regulatory status, given by agencies like the U.S. Food and Drug Administration of drugs/therapies developed for rare diseases or conditions. The benefits of orphan drug designation can be significant:  

Financial Incentives

Sponsors of orphan drugs, including gene therapies, may be qualified for tax credits for clinical testing and can waive the human drug application fee upon submission of a marketing application. 

Exclusivity for Orphan-Drug Designation 

Orphan-drug designation offers seven years of market exclusivity if the eligibility criteria are satisfied. No similar product will be approved for that rare disease or condition in that period. 

Market Access 

Orphan drug designation can fast-track the regulatory process for market access, accelerating life-saving treatments to come on the market. 

However, orphan designation is not an easy thing to get. It involves a satisfactory explanation of how the product would be used for a rare disease or condition and, in some instances, the clinical benefit of the product over an existing treatment. 

Orphan Drug Designation Process for Gene Therapy Products

Eligibility Criteria

A gene therapy product is considered an orphan drug if it is intended for the treatment of a rare disease or condition. Rare diseases are those that affect fewer than 200,000 individuals in the United States.

Scientific Rationale

Sponsors should provide sufficient scientific rationale to allow for the establishment of a medically plausible basis that would support expectations regarding the effectiveness of the gene therapy product for treating the rare disease. This should be well-documented, based on solid preclinical data, and complemented by available clinical data. 

Filing an application

A sponsor must apply to the OOPD for orphan drug designation in accordance with the procedures in 21 CFR 316.20. An application for orphan drug designation may be submitted to FDA at any time before the submission of an application for marketing. 

Addressing Clinical Superiority

When a gene therapy product is the same as an approved drug for the same use or indication, sponsors of such products must provide a reasonable hypothesis that their products are clinically superior to the existing treatment. Clinical superiority may be based on greater efficacy, greater safety, or major contribution to patient care. 

Interpreting the Sameness of Gene Therapy Products

The products of gene therapy are thus some critical challenges in obtaining orphan drug designation, and determining the “sameness” of gene therapy products for the same use or indication will be one of the critical challenges. 

Transgenes and Vectors 

If two gene therapy products express different transgenes for the same rare disease, they are considered different drugs even if they use the same vector. 

Different vectors

When different vectors are employed, two gene therapy products will typically be considered different drugs, regardless of whether they express identical or various transgenes. Vector selection can affect the following: Tropism Immune response avoidance or even the potential for insertional mutagenesis. 

Minor Differences

Such minor differences in transgenes and vectors, including polymorphism, would not provide a different drug classification. Determination of minor differences is on a case-by-case basis. 

Additional characteristics

For two gene therapy products to be considered the same if they express the same transgene, using the same vector, the decision could depend on other characteristics of the final product that impart therapeutic effect. These may include regulatory elements such as promoters, enhancers, or splicing elements, cell type transduced. These issues are also considered on a case-by-case basis. 

Conclusion 

Besides this, the designation as an orphan drug promises enormous advantages to several gene therapy products developed in the interest of patients suffering from a rare disease or condition. However, such complex regulatory pathways will demand a broad scientific and regulatory landscape for reaching a decision on “sameness.” 

Our regulatory consulting firm realizes the need for regulatory consultation that could advise the sponsor of the gene therapy product in the process for the designation of an orphan drug. 

By harnessing the power of regulatory knowledge, we help develop and approve gene therapy products with the potential to transform the lives of patients with rare diseases. 

To learn how we can help your organization, please contact BioBoston Consulting