It’s officially Rare Disease Week, which is being celebrated from February 22nd until March 2nd. Every year, we take this week to to recognize, support, raise awareness, and advocate for the rare disease community. One goal of this week is to push for the development of rare disease treatments, which many patient populations are in desperate need of.

To celebrate the victories we’ve already won within the rare disease treatment landscape, Patient Worthy is compiling a list of the treatments that the FDA approved back in 2021.

Rare Disease Treatments Approved in 2021

Throughout the course of last year, 60 drugs were granted approval by the FDA. The majority of these drugs (50 medications) were approved by the Center for Drug Evaluation and Research (CDER), while the remaining ten received approval from the Center for Biologics Evaluation and Research (CBER).

The CDER published a report reviewing the approved drugs. Here are some of the most important details:

• More than half (54%) of the CDER-approved drugs were first-in-class
  • Lupus and lupus nephritis both saw new treatment options from this category
  • Other disease states that saw approved treatments last year include lung cancer, HIV, and endometrial cancer, among others
• The CBER approvals include five vaccines and CAR-T cell therapies for cancers like multiple myeloma and lymphoma
  • The first-ever COVID-19 vaccine falls into this category
• 76% of CDER-approved therapies were approved in the United States before any other country
• 74% of CDER-approved therapies utilized at least one expedited program, while 28% were placed onto the accelerated approval pathway
• The Prescription Drug User Fee Act (PDUFA) continued to help patients access treatments faster
• Many treatments saw continued research that led to expanded indications
  • Many indications grew to include pediatric patients
    • Pediatric patient populations that benefitted from this include sickle cell disease patients, cystic fibrosis patients, hepatitis C patients, HIV patients, and more
• 52% of CDER-approved drugs are indicated for the treatment of rare diseases
  • Rare diseases that saw treatments approved last year include rare cancers, Pompe disease, molybdenum cofactor deficiency type A, rare blood diseases, and more

Looking Forward

While the number of FDA-approved treatments changes from year to year, it was very encouraging to see that this number did not drop significantly during the ongoing COVID-19 pandemic. Hopefully, there is continued commitment to the development of treatments for rare disease as we move forward into 2022.

Find the source article here.