FDA Orphan Designation →
- The Coté team offers unparalleled expertise and efficiency in constructing the Orphan Drug designation application. We write the entire application, including the epidemiology and scientific rationale sections. A first draft takes our team two to three weeks to prepare. Most applications are submitted within three weeks of our team initiating work.
EMA Orphan Designation →
- We partner with our clients to decide whether an optional pre-submission meeting will optimize their likelihood of success. Additionally, we write the entire EMA Orphan Designation application and submit on the designated submission date. Our team will handle the management of the extensive translations required by EMA for submission. Because Coté has a European entity we are able to submit on behalf of those clients who do not possess such an entity.
Strategic Reviews →
- Our regulatory experts suggest to the client a product development strategy, while taking the current regulatory environment into consideration, to optimize an orphan drug development pathway. We perform an in-depth analysis of the epidemiology and the therapeutic area. Our strategic reviews provide a feasibility assessment of a specific molecule and disease state or a review of the client’s entire pipeline to discover and pursue all “orphanable” pathways.
Meeting Request & Briefing Package →
- We lead FDA Type A/B/C meetings, meetings with the OOPD, as well as Scientific Advice in the EU. Our expert team has direct experience with all FDA review divisions, with valuable knowledge of how to develop successful proposals to regulators.
Breakthrough Therapy Designation →
- Our team has had great success in writing breakthrough therapy designations. Our depth of knowledge and experience in working with each review division informs the strategy of each application.
RMAT Designation →
- Our regulatory staff are well-versed in the unwritten rules of the FDA, and have had great success helping our client’s products achieve breakthrough status, among other designations
Priority Review Voucher →
- We are the largest submitter of priority review designation applications. We understand the challenge of these applications, and in particular, the level of scrutiny given to the epidemiology section.
IND Filings →
- We prepare, submit and maintain INDs. We work with many clients to streamline their strategy and work towards Orphan Designations, Pre-IND meetings, and IND submissions. Our team has a unique skill set that can generate successful submissions, in a rapid and expert manner.
- We stay with our clients from the lab to the market. We have strong experience in preparing marketing applications, as well as participating on the review of applications from inside the FDA.
What is an Orphan Drug?
Orphan drugs are medicinal products intended for diagnosis, prevention, or treatment of rare diseases or disorders. A “rare” disease is defined as a condition that affects fewer than 200,000 people in the U.S. (5 per 10,000 in Europe) or the prevalence is greater but the drug manufacturer is not expected to recover the costs of developing and marketing the treatment.
Why Apply for an Orphan Drug Designation?
Obtaining orphan designations is one of the first and most fundamental steps in developing a drug that treats a rare disease. Benefits include:
- Seven years of marketing exclusivity
- Tax credits
- PDUFA Fee exemption
- Regulatory support from the Office of Orphan Product Development (OOPD)
- Extraordinary interest from the investment community in developing orphan drug products
- Access to the OOPD grants program
Obtaining orphan designation greatly strengthens the business case and investment thesis for early stage drugs, providing the momentum to enter the clinic.