FDA watch: Novel drug approvals in 2020

The coronavirus pandemic may have upended millions of American lives and stalled the global economy, but that hasn’t prevented the FDA Center for Drug Evaluation and Research (CDER) from doing a vital job during the health crisis: Ensuring the safety and efficacy of drugs. 

The agency has approved 48 novel drugs in 2020, which is quite the feat amid a pandemic and even more impressive when stacked up to previous years. The FDA approved 48 novel drugs in 2019, 59 in 2018, 46 in 2017, and 22 in 2016.  

Therapies approved this year will treat dozens of rare, common, deadly, and benign conditions, from Duchenne muscular dystrophy to high cholesterol, and non-small cell lung cancer to head lice. 

The agency also carefully monitored approved therapies and, when new evidence revealed that the risk of a drug or ingredient outweighed its benefits, pulled them from the market. This has happened hundreds of times this year. 

It can be difficult to keep track of all FDA developments. But have no fear—we’re here to bring you the most notable approvals of 2020.

Palforzia for peanut allergy

On January 31, the FDA approved Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp, formerly AR-101] for the treatment of peanut allergy in children. The approval was hotly anticipated because it is the first-ever drug for the condition, and because its market availability will make a tremendous difference to the roughly 1 million children in the United States with peanut allergy—a condition that can cause life-threatening reactions and for which there is currently no cure.

Palforzia is a powder made from peanuts that helps patients escalate dosing over time—a treatment tactic known as an oral food challenge. Its effectiveness was supported by studies across the United States, Canada, and Europe, conducted on roughly 500 people with peanut allergies. Among those who took Palforzia, 67.2% tolerated a 600 mg dose of peanut protein in the oral food challenge, compared to just 4% who received placebo. 

Trodelvy for metastatic triple-negative breast cancer

In its first few FDA tests, Trodelvy (sacitizumab govitecan-hziy) earned orphan drug, fast track, and breakthrough therapy designations. These markers highlighted the fact that the FDA believed the drug would fill an unmet medical need and demonstrate a substantial improvement over available breast cancer therapies. On April 22, the FDA granted accelerated approval to Trodelvy for adult patients with metastatic triple-negative breast cancer (mTNBC) who received at least two prior therapies for metastatic disease.

Approval was based on data from IMMU-132-01, a multicenter, single-arm trial enrolling 108 patients with mTNBC. The trial’s primary outcome measures were investigator-assessed overall response rate (ORR) and response duration. The ORR was 33.3% and median response duration was 7.7 months. 

The recommended dose for this drug is 10 mg/kg administered by IV infusion on days 1 and 8, and every 21 days after until there is disease progression or an unacceptable level of toxicity.

Tauvid for Alzheimer disease imaging

The medical community has eagerly anticipated a new therapy for Alzheimer disease for decades. While Tauvid (flortaucipir F18)—a drug used to image tau pathology in patients being evaluated for Alzheimer disease—isn’t the long-awaited Holy Grail, it moves us closer to earlier diagnosis, better treatment, and the potential for novel therapies for the condition. The drug was approved on May 28.

When Tauvid is administered intravenously, it binds to sites in the brain associated with tau protein misfolding, the hallmark of Alzheimer disease. PET scans can then help identify the presence of tau pathology and facilitate earlier diagnosis of Alzheimer, a disease that ranks among the top 10 leading causes of death in the United States, affecting an estimated 5 million Americans.

Nurtec ODT for acute migraine

The treatment landscape for migraine has steadily improved in recent years, but many patients remain underwhelmed by available options and their routes of administration, which sometimes involve injection. The situation improved on February 27, when the FDA approved Nurtec orally disintegrating tablets (ODT) (rimegepant) for acute treatment of migraine in adult patients. 

Nurtec ODT was the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in a fast-acting, orally disintegrating tablet. In trials, a single 75 mg oral dose provided fast pain relief and returned patients to normal function within 1 hour, with many patients seeing sustained efficacy for 48 hours. What’s more, 86% of patients treated with a single dose of the drug didn’t need to use a migraine rescue medication within 24 hours.

Nexletol for cholesterol and cardiovascular disease

Statins remain the go-to treatment for high cholesterol, but for some, they are not effective enough. These patients can now turn to Nexletol (bempedoic acid), which, as of February 21, became the first FDA-approved drug in a new class of cholesterol medications, known as ATP citrate lyase (ACL) inhibitors. This drug class works by preventing a part of the cholesterol production process in the liver that’s different from the way statins work. 

The drug is approved to be taken alongside statins to further lower cholesterol and reduce the risk of heart attack and stroke in situations where statins don’t go far enough. Nexletol is available in 180 mg tablets to be taken once daily.

Zeposia for multiple sclerosis  

Zeposia (ozanimod) was approved on March 25 after clinical trials demonstrated that it could effectively reduce annualized relapse rates (ARR) in adult patients with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. The drug is taken orally, twice a day.

In the phase 3 RADIANCE and SUNBEAM trials, Zeposia demonstrated a relative reduction in the AAR of 48% through one year and 38% through two years vs the anti-inflammatory multiple sclerosis treatment Avonex, which is among several currently available first-line treatments for MS. Zeposia outperformed Avonex in its ability to reduce the number and size of brain lesions in trial participants.