March 10, 2020

Priority Review Voucher Expires?

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 In July 2019 the FDA released revised draft guidance describing their thinking on the Rare Pediatric Disease Priority Review Voucher program, originally set forth in Section 529 of the FD&C Act and revised by the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.
June 4, 2018

It’s Time for #BIO2018!

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.
October 4, 2017

Orphan Insights, Part 3: Breakthrough Therapy Designation

A Breakthrough Therapy (BTD) is an FDA designation that was introduced in 2012 as a means to permit the FDA to grant priority review to a drug candidate under development for a serious or life-threatening disease, when clinical evidence indicates the therapy offers substantial treatment advantages over existing therapies for patients.
September 25, 2017

Orphan Insights, Part 2: The PIP

Question #1 – What are the disadvantages, if any, of filing for Orphan Drug Designation? There are really no true disadvantages in filing for orphan designation. Question #2 - When is the right time to submit a PIP? This depends on a lot of variables. In general, it can be submitted prior to initiating trials and up until initial proof-of concept trials, definitely before pivotal or confirmatory trials.