What Novel Drugs Did the FDA Approve in 2021?

Written by Timothy Aungst, PharmD | Reviewed by Joshua Murdock, PharmD Published on February 17, 2022

Key takeaways:

• The FDA approved 50 novel (new) drugs in 2021. This is similar to the number of novel drugs approved in 2020.
• Over half of the novel drugs approved in 2021 were for rare diseases. These are diseases that affect less than 200,000 people in the U.S.
• There were also many first-in-class therapies approved in 2021. This means they work differently than other medications on the market.

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Every year, medications are approved to treat various health conditions in the U.S. This includes novel (new) medications with active ingredients that haven’t been approved before.

The Center for Drug Evaluation and Research (CDER) is the part of the FDA responsible for overseeing many of the new drug approvals in the U.S. In a recent report, CDER announced it had approved 50 novel medications in 2021. Of these, over half are first-in-class and/or are used to treat rare diseases.

Here, we’ll review notable approvals from 2021, as well as what we might expect in 2022.

What are novel drugs?

The FDA evaluates and approves many medications every year. Some of the approvals are for new generics or different dosage forms (e.g., capsules or liquids). 

But other approvals are for new and innovative medications that are different from those currently on the market. These medications are referred to as novel drugs.

If these novel drugs fulfill an unmet medical need, they may also qualify for special FDA designations. This helps speed up the review or approval process. These designations include Fast Track, Breakthrough Therapy, and Priority Review. Some medications also qualify for Accelerated Approval. This helps get important treatments to people who need them faster.

CDER has been releasing novel drug approval reports since 2011. This is to highlight new innovative treatments that could have a positive impact on patient care in the U.S.

Examples of novel drugs approved in 2021

CDER approved 50 novel drugs in 2021. Most received one or more FDA designations to expedite their review and/or approval.

Some examples of these novel drugs include:

• Cabenuva (cabotegravir and rilpivirine): a long-acting injectable medication used to treat HIV
• Qelbree (viloxazine): a new non-stimulant medication to treat ADHD in children and adolescents ages 6 to 17
• Verquvo (viricuguat): a new medication that can help lower the risk of death and hospitalization in people with advanced heart failure
• Zegalogue (dasiglucagon): an injectable medication that can help save people who have dangerously low blood sugar levels (hypoglycemia)

Examples of novel drugs approved for rare diseases in 2021

Novel drugs can also be further broken down into different categories. One of these categories is “orphan drugs,” or medications used for rare diseases. Rare diseases are those that affect less than 200,000 people in the U.S. In 2021, the FDA classified 26 of the 50 drugs as orphan drugs. 

Examples of these medications include:

• Besremi (ropeginterferon alfa-2b-njft): an injectable medication used to treat a blood disease called polycythemia vera (results in too many red blood cells in your blood)
• Fexinidazole: the first all-oral treatment for sleeping sickness, which is caused by parasites transmitted by tsetse flies in parts of Africa 
• Lumakras (sotorasib): an oral medication for a certain type of non-small cell lung cancer that was originally thought to be resistant to medications
• Skytrofa (lonapegsomatropin-tcgd): an injectable medication that’s the first once-weekly treatment for children with growth hormone deficiency

What are first-in-class drugs?

CDER classifies first-in-class drugs as the first of their kind to appear on the market. In other words, there haven’t been other medications that work the way they do until now. First-in-class medications can offer new solutions for people with conditions that haven’t responded well to current options.

Examples of first-in-class drugs approved in 2021

Over half of the 50 novel drug approvals were first-in-class drugs.

 Some examples include:

• Aduhelm (aducanumab-avwa): the first new Alzheimer’s treatment since 2003, and the first to treat the underlying disease process
• Evkeeza (evinacumab-dgnb): an injectable medication that can be added to other cholesterol-lowering medications for genetic high cholesterol
• Kerendia (finerenone): an oral medication that helps lower the risk of kidney and heart problems from diabetes and chronic kidney disease (CKD)
• Saphnelo (anifrolumab-fnla): the first new medication to treat systemic lupus erythematosus (SLE) in 10 years
• Tezspire (tezepelumab-ekko): an add-on injectable medication for people ages 12 and older with severe asthma
• Leqvio (inclisiran): a twice-yearly injectable medication used to lower LDL (“bad”) cholesterol

What novel drugs could be approved in 2022?

It’s likely that we’ll see a continued push for more novel drug approvals in 2022. So far, several new medications have been approved. One example is Quviviq (daridorexant), a new insomnia treatment. 

Other medications are still under review. For example, Jardiance (empagliflozin) received breakthrough therapy designation for heart failure with preserved ejection fraction (HFpEF) in 2021. If approved in 2022, it would be the first medication able to treat both types of left-sided heart failure.

Other potential novel drug approvals in 2022 could include:

• Tapinarof: a novel steroid-free topical cream for plaque psoriasis
• Deucravacitinib: first-in-class oral medication for moderate to severe plaque psoriasis
• Mavacamten: novel oral medication for obstructive hypertrophic cardiomyopathy, the most common inherited heart disease
• Tirzepatide: a first-in-class injectable medication for Type 2 diabetes
• Ganaxolone: a first-in-class treatment for a rare genetic type of epilepsy

The bottom line

In 2021, we saw many novel medications approved by the FDA. They included treatments for numerous conditions, ranging from autoimmune disorders and cancer to Alzheimer’s disease.

Over half of the novel drug approvals were for rare diseases. This may be highly beneficial for thousands of people across the U.S. who may not have had access to adequate treatments yet. Looking ahead to 2022, there are several notable novel drugs that could be approved. Check back with GoodRx for updates.