Rare Disease Drugs Led the Way for 2020

According to the FDA, there were 53 novel drug approvals in 2020, with 32% receiving FDA fast track status, 57% receiving priority review status, and 23% obtaining accelerated approval.  This count does not include vaccines, plasma products, or gene therapy products.  With so many 2020 drug approvals receiving priority review, it is likely that many of the drugs could provide a significant advance in medical care.

Many of the newly approved agents that received priority review status are indicated for rare disease states.  Oncology approvals continue to outpace approvals for other disease states, with approximately 37% of drugs approved in the oncology space, whether for diagnosis or treatment.

Six new infectious disease agents were approved, which is a positive step in developing new therapies to combat malaria, Ebola, HIV-1, COVID-19, and a rare parasitic disease known Chagas disease.  Artesunate has been approved by the FDA to treat severe malaria in the U.S.  Ebanga and Imazeb were approved to treat Ebola, while Rukobia was approved to treat HIV-1.  One treatment for COVID-19, Veklury, received fast track and priority review due to the Coronavirus pandemic. The FDA approved Lampit, a treatment for pediatric patients with Chagas disease, a rare parasitic disease, which if left untreated, can lead to congestive heart failure.

What does 2021 hold?  PHSL highlights three top trends to watch for in this year’s drug approval space:

  1. COVID-19 – Will additional therapeutics to treat infections be approved?
  2. Alzheimer’s – Will the drought for new treatments end in 2021?
  3. Delays – The pandemic has already impacted trials, inspections, and launches in 2020. Will more of the same persist in 2021?

A full listing of 2020 approvals, by approval type, is shown in the chart below.


Posted January 2021

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