CDER notches uptick in novel drug approvals in 2023

Editor's note: This article was updated on 11 January 2024 to correct a typo.

The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) issued a report on the 55 novel drugs approved by the center in 2023, as well as other important center actions such as expansions on use or patient populations for previously approved therapies and drugs approved in new dosage forms and formulations.
 
The number of new drugs approved by CDER last year was notably higher than 2022, which saw 37 approvals, and the highest year for novel approvals since 2018, which saw 59 approvals. From 2014 to 2023, CDER has averaged approximately 46 novel drug approvals per year.
 
The 2023 “drug approvals collectively target a wide range of diseases and conditions,” said CDER Director Patrizia Cavazzoni, noting that many “of the 2023 approvals are for patients with few or no treatment options, including those with rare diseases,” while other “drug approvals offer improvements in effectiveness, safety, or ease of use compared to standard-of-care therapies.”
 
Cavazzoni highlighted that CDER “approved the majority of the 2023 therapies on or before their goal dates, or congressionally authorized agreements with industry” and approved most of these drugs “in the US before our regulatory counterparts did so in other countries.”
 
In fact, according to the report, 35 of the 55 novel drugs cleared in 2023, or 64 percent, were initially approved in the US before being approved elsewhere.
 
And Cavazzoni singled out CDER’s efforts in the opioid overdose space.
 
“In 2023, CDER took actions to make three opioid overdose reversal drugs available without a prescription, which can increase access to life-saving therapies,” she said. “We also approved new dosage forms of drugs that reverse opioid overdose and treat opioid use disorder,” Cavazzoni continued, noting that her center’s “efforts align with FDA’s Overdose Prevention Framework, which aims to prevent drug overdose and reduce overdose-related death.”
 
CDER also said it met or exceeded its Prescription Drug User Fee Act (PDUFA) goal dates for 49 of the 55 approvals (89%). Some of these drug approvals in 2023 were delayed due to still ongoing COVID-19-related foreign travel restrictions, which continued to hinder onsite inspections within user fee review timelines.
 
The report noted that CDER approved 46 of the 55 drugs (84%) on the first cycle in 2023, which “differs from when CDER initially is unable to approve a drug because information in the application does not support approval … the sponsor resubmits the application with additional information, starting another review cycle that may lead to drug approval.”
 
Notably, CDER approved five biosimilars last year, including three biosimilars for reference therapies that did not previously have a corresponding biosimilar. To date, this brings CDER’s total approved biosimilars to 45, for 14 total reference products.
 
In another finding, the report noted that 36 of the 55 novel drug approvals, or 65 percent, used at least one FDA expeditated development and review program: fast track designation, breakthrough therapy designation, priority review designation, or accelerated approval.
 
In addition, 20 of the 55 novel drugs approved, or 36 percent, in 2023 were deemed first-in-class, meaning they contain a mechanism of action different from existing therapies. Such novel drugs approved in 2023 identified as first-in-class are: Daybue (trofinetide), Defencath (taurolidine/heparin), Fabhalta (iptacopan), Filspari (proteinuria), Filsuvez (birch triterpenes), Jesduvroq (daprodustat), Joenja (leniolisib), Lamzede (velmanase alfa-tycv), Miebo (perfluorohexyloctane), Ogsiveo (nirogacestat), Paxlovid (nirmatrelvir/ritonavir), Qalsody (tofersen), Rivfloza (nedosiran), Skyclarys (omaveloxolone), Sohonos (palovarotene), Talvey (talquetamab-tgvs)), Truqap (capivasertib), Veopoz (pozelimab-bbfg)), Veozah (fezolinetant), and Xdemvy (lotilaner).
 
In terms of therapies for rare diseases, the report said that 28 of CDER’s 55 novel drug approvals in 2023, or 51 percent, received orphan drug designation, given to drugs that treat conditions affecting fewer than 200,000 people in the US who have few or no treatment options available.
 
Report