Special Applications

Orphan Drug Designation (ODD)

Orphan Drug Designations are the FDA’s program to incentivize development of new therapies for the treatment of rare diseases (generally diseases or conditions affecting fewer than 200,000 people in the US). The FDA grants ODD for drugs/biologics that prevent, diagnose or treat a rare disease / condition. That designation provides benefits such as tax credits for qualified clinical trials, exemption from user fees, and potentially seven years of market exclusivity following approval.

ODD submissions need to be carefully crafted, requiring input from a cross functional team with experts in pharmacology, epidemiology, and various regulatory affairs disciplines (nonclinical, CMC, medical writing & strategy) and as a result they can be quite complex. To be successful, sponsors need to prove their indication meets the rare population criteria, provide clinical data to corroborate their request, and demonstrate a clear mechanism of action (MOA). Successful ODD applications can also vary significantly with some focusing on the MOA without clinical data, and others targeting a subset of a condition.

The best way to guarantee a successful ODD is to work with experts who have successfully submitted ODDs to FDA, and have a deep understanding of their requirements. To date Aleon has successfully completed more than 20 ODDs with the FDA, several of which are now fully approved drugs with market exclusivity for years to come. Reach out today to see if an Orphan Drug Designation is right for your product!

Fast Track Designation

Fast Track Designation is an FDA process designed to facilitate the development and expedite the review of drugs to treat serious conditions and unmet needs in the US, of which there are many. Broadly, the FDA expects qualifying drugs to fill an unmet need where no therapy exists, or to be an improvement compared to available therapies by having a positive impact on survival, day-to-day well being, or preventing the underlying condition from worsening. Receiving the Fast Track Designation will make sponsors eligible for more frequent meetings/ written communications with the FDA, and potentially make them eligible for Accelerated Approval and Priority Review or Rolling Review. To properly benefit from a Fast Track Designation Aleon recommends applying early in the drug development process, and keeping experts on hand to handle the frequent communication and meetings with the FDA.

Breakthrough Therapy Designation

Breakthrough Therapy Designation is for novel drugs that represent a significant improvement over the existing therapies on any number of clinically significant endpoints (such as serious symptoms of a disease, or an impact on irreversible morbidity or mortality). A successful designation entitles sponsors to more frequent meetings/written communication with the FDA, and potentially makes them eligible for Accelerated Approval and Priority Review or Rolling Review. In addition the FDA can provide significant additional guidance on an efficient drug development program and involve FDA senior managers to further expedite the program.

Breakthrough Therapy Designation is very similar to Fast Track Designation, and from a distance, sponsors may not understand the difference. The main difference is the type of data sponsors will need to qualify - Fast Track can rely on nonclinical data, whereas Breakthrough Therapy needs to rely on clinical data. There are more subtle differences for these programs based on the development strategy of a given product, to understand how to proceed Aleon recommends consulting with our team who has successfully submitted many of these applications!

Accelerated Approval

Accelerated Approval differs from the other accelerated designations because it is an approval pathway rather than a designation. It allows therapies to be conditionally approved based on a biomarker or intermediate clinical endpoint that strongly indicates clinical benefit. Typically these agreements occur close to NDA/BLA approval and require additional confirmatory studies after receiving accelerated approval. Accelerated Approval can be part of a successful drug development strategy, but if handled improperly can lead to longer delays or the product’s removal from the market. Additionally, the approval will require sponsors' regulatory affairs team to have an understanding of special labeling requirements and surrogate endpoints. When considering an Accelerated Approval, sponsors should consult with experts early in the process to determine if it is the right pathway for them.

Regenerative Medicine Advanced Therapy Designation (RMAT)

Regenerative Medicine Advanced Therapy Designation is a special designation for therapies which meet three specific criteria: it is a regenerative cell therapy, it treats a serious or life-threatening condition, and preliminary clinical evidence indicates it can treat such a condition. The benefits of a successful RMAT designation are similar to Breakthrough Therapy, namely increased communication with the FDA and early meetings to discuss potential surrogate or intermediate endpoints. The FDA has released several new guidances on the relevant definitions, and the designation must be submitted concurrently with an IND or as an amendment to an existing IND. RMATs can be tricky even for an experienced sponsor, so Aleon recommends consulting with experts before committing to this strategy.

Priority Review Designation

The last expedited program at the FDA is Priority Review. Unlike the other programs, Priority Review is requested by sponsors at the time of NDA/BLA submission, and can reduce the necessary review time at the FDA by four months. To qualify sponsors need to demonstrate a significant improvement in treatment/diagnosis/prevention, a significant reduction of a treatment-limiting drug reaction, enhancement in patient compliance (that leads to an improvement of serious outcomes) or increased safety/effectiveness in a new population subset. Priority Review may be expressly requested by the sponsor and, given the potential upside, is best done with a team of experts helping to support your claim. If you think your product may be eligible, reach out today and find out!

Aleon Pharma International, Inc. is a one-stop, full-service regulatory affairs consulting firm. When you work with Aleon, we evaluate your program and carefully assemble a dedicated and experienced project team that is tailored to achieve your specific project goals. We have dedicated project teams in both the US and China who are specialized in Regulatory Strategy Development, Regulatory Writing, Chemistry, Manufacturing and Controls, Nonclinical Strategy, Clinical Development, eCTD Publishing, and submissions to the FDA, NMPA, EMA, and other regulatory agencies.

Aleon has worked with many sponsor companies over the years, and our team has developed streamlined processes that enable us to effectively provide high-quality services that help you achieve approval for your innovative drug candidates.