Only Orphans Cote
Only Orphans Cote is a specialized orphan drug consulting firm led by the former Director of the FDA Office of Orphan Products Development. The company provides expert services in obtaining FDA and EMA orphan drug designations, regulatory strategies, and support throughout the drug development process to help clients bring their rare disease drugs to market. With a team including former FDA reviewers, they leverage extensive regulatory experience to maximize the chances of approval and market success.
Industries
Nr. of Employees
small (1-50)
Only Orphans Cote
625 Massachusetts Ave, Cambridge MA 02139
Services
Drafting complete orphan designation applications for US and EU regulatory submissions, including prevalence and medical rationale support.
Program assessment and creation of regulatory roadmaps to optimize development, incentives use, and regulatory interactions.
Preparation of briefing packets, meeting strategy, and post-meeting follow-up documentation for agency interactions.
Review, analysis, and authorship of IND applications to enable initiation of human clinical studies.
Preparation of designation requests and supporting evidence for Breakthrough Therapy, Fast Track, Accelerated Approval, and RMAT.
Preparation of grant submissions for clinical trials and natural history studies and alignment with reviewer expectations.
Drafting complete orphan designation applications for US and EU regulatory submissions, including prevalence and medical rationale support.
Program assessment and creation of regulatory roadmaps to optimize development, incentives use, and regulatory interactions.
Preparation of briefing packets, meeting strategy, and post-meeting follow-up documentation for agency interactions.
Review, analysis, and authorship of IND applications to enable initiation of human clinical studies.
Preparation of designation requests and supporting evidence for Breakthrough Therapy, Fast Track, Accelerated Approval, and RMAT.
Preparation of grant submissions for clinical trials and natural history studies and alignment with reviewer expectations.
Expertise Areas
- Orphan drug regulatory affairs
- FDA and EMA orphan designation processes
- Regulatory strategy and meeting preparation
- IND and early clinical regulatory support
Key Technologies
- Preclinical animal model evidence
- Clinical trial design for rare diseases
- Surrogate endpoint strategy
- Regulatory dossier and submission preparation
News & Updates
World Orphan Drug Congress 2025
An upcoming biotech conference in Boston, MA, from April 22-24, 2025, where the OOC team will be present.
LSX World Congress USA
A biotech event in Boston, MA, scheduled for September 16-17, 2025.
ASGCT 2025
American Society of Gene & Cell Therapy conference in New Orleans, LA, from May 13-17, 2025.
RAPS Convergence
Regulatory Affairs Professionals Society conference in Pittsburgh, PA, from October 7-9, 2025.
BIO International 2025
Major biotech conference in Boston, MA, from June 16-19, 2025.
Bio Japan 2025
Biotech event in Yokohama, Japan, from October 8-19, 2025.
World Orphan Drug Congress 2025
An upcoming biotech conference in Boston, MA, from April 22-24, 2025, where the OOC team will be present.
LSX World Congress USA
A biotech event in Boston, MA, scheduled for September 16-17, 2025.
ASGCT 2025
American Society of Gene & Cell Therapy conference in New Orleans, LA, from May 13-17, 2025.
RAPS Convergence
Regulatory Affairs Professionals Society conference in Pittsburgh, PA, from October 7-9, 2025.
BIO International 2025
Major biotech conference in Boston, MA, from June 16-19, 2025.
Bio Japan 2025
Biotech event in Yokohama, Japan, from October 8-19, 2025.