In early January, CDER released its report on the 59 new drug therapies approved in calendar year 2018. Among the new drugs approved last year are treatments for rare diseases like phenylketonuria, the first in a new class of drugs to treat non-responding HIV-1 patients, and a host of new and more selective cancer treatments. Some items to note include:

• The first therapies in a class of drugs targeting the abnormal RNA that is the root cause of hereditary transthyretin amyloidosis,
• The first drug ever to treat smallpox under the Animal Rule, which allows efficacy findings from well controlled animal studies to be used when it is not feasible or ethical to conduct human trials. 
• Three new drugs from the class calcitonin gene-related peptide receptor (CGRP-R) antagonists, all for the prevention of migraine.
• An innovative new generating system for radio imaging agent Technetium-99m that does not rely on enriched uranium as the feedstock for its supply chain. This innovation improves the supply of this important radioisotope and enhances global safety.

Twenty-four of the new drugs received the Fast Track designation, and 14 were designated Breakthrough Therapies, which receive all the features of the Fast Track program plus more intensive guidance from the Center’s reviewers. You can read more about the new therapies approved in previous years at the New Therapies website.

Text Copyright © 2019 Katrina Rogers