When Dr. Martin Makary took his seat as FDA Commissioner earlier this year, his early remarks signaled a fresh era for rare disease and gene therapy developers. From promising to “customize the approval process to the condition” to voicing support for faster reviews in ultra-rare populations, Makary struck an optimistic tone that resonated with RareMoon’s … Read more
The second half of 2025 is shaping up to be a pivotal period for advanced therapies, with the FDA poised to decide on a wave of gene and cell therapies. The outcomes of these reviews will offer critical insights into the future of Accelerated Approval for gene therapies and signal how new FDA leadership might … Read more
Despite FDA staffing concerns, our experience has been steady—fast responses, consistent support, and strong collaboration continue.
Navigate regulatory uncertainty with strategy, early engagement, and thoughtful communication to build trust, reduce risk, and drive rare disease programs forward.
RareMoon exists to navigate rare disease drug development, overcoming regulatory hurdles to bring life-changing therapies to patients. #RareDiseaseDay
RareMoon expert Mette Due Theilade Thomsen to lead DIA Global’s virtual Paediatric Investigation Plans (PIP) training in November, offering key regulatory insights and practical skills.
The FDA’s RPD PRV Program has led to 569 RPD designations in 10 years, boosting treatments for rare diseases like Duchenne muscular dystrophy, neuroblastoma, and sickle cell.
Time flies. In July 2019 the FDA released revised draft guidance describing their thinking on the Rare Pediatric Disease Priority Review Voucher program, originally set forth in Section 529 of the FD&C Act and revised by the Advancing Hope Act of 2016.
It’s officially day one of #BIO2018 for Boston, the industry, and RareMoon. Co-founders, Chris and Sabrina, will be taking on #BIO2018 together this year to divide, conquer, and make the best of the week. This year has already been a fantastic year for RareMoon and our growth both as a company and in the intimate support we’ve provided to global biotech & pharma companies developing orphan medicines.
