New Drug Approvals in 2024: How Did The Industry Fare?

How did the industry fare with new drug approvals in 2023? What new drugs were approved by FDA and were approvals in pace with prior years? What was the mix between small molecules and biologics, and which companies and products made the mark? DCAT Value Chain Insights sums it up.

How did the industry fare with new drug approvals in 2024? What new drugs were approved by FDA and were approvals in pace with prior years? What was the mix between small molecules and biologics, and which companies and products made the mark? DCAT Value Chain Insights sums it up.

By Patricia Van Arnum, Editorial Director, DCAT, pvanarnum@dcat.org

New drug approvals keep pace
In 2024, the US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 50 new molecular entities (NMEs) and biological therapeutics, compared to 55 new drugs approved in 2023, keeping pace with recent trends of reaching at least 50 new drug approvals. The 50 new drugs approved by FDA’s CDER in 2024 marked the fifth year out of the past seven years with new drug approvals reaching or exceeding 50 (2018–2024), with new drug approvals only dipping below 50 twice (in 2019 with 48 new drug approvals and 2022 with 37) (see Figure 1 for historical trends).

Small/mid-sized companies dominated new drug approvals in 2024. Of the 50 new drugs approved in 2024, 14, or 28%, were from large companies (defined as the top 25 bio/pharma companies based on 2023 revenues and one large medical technology company with approval of a diagnostic agent), and 36, or 72%, were from mid-sized or small companies (see Table I at end of article for the full listing of new drug approvals by FDA’s CDER in 2024).

Small molecules versus biologics
In looking at the product mix of new drug approvals, of the 50 new drug approvals by FDA’s CDER in 2024, 34 or 68%, were small molecules (31 drugs and three diagnostic/imaging agents), and 16, or 32%, were biologics. That is on par with the mix in 2023, when FDA’s CDER approved 17 new biological therapeutics, which represented 31% of new drug approvals, and 38 small-molecule products or 69% of new drug approvals. However, small-molecule approvals in 2024 and 2023 dipped somewhat and were below recent historical levels when small-molecule drug approvals averaged 75% of new drug approvals between 2019 and 2021, with 2022 being an outlier with only 37 new drug approvals overall and only 59% being small molecules (see Figure 2 for historical trends).

Small-molecule new drug approvals in 2024
Small/midsized companies also dominated small-molecule drug approvals. Of the 34 small-molecule drug approvals in 2024, only six, or approximately 18%, were from the large companies (five drugs and one radioactive diagnostic drug). In contrast, small-to-mid-sized companies accounted for 28 small-molecule new drug approvals (26 drugs and two imaging/diagnostic agents), or 82%, of the small-molecule approvals by FDA’s CDER in 2024. AstraZeneca, Bristol-Myers Squibb, Gilead Sciences, Johnson & Johnson, and Roche, each had one small-molecule drug approvals as did GE HealthCare for a radioactive diagnostic drug. Small-molecule approvals by the large companies were:  

  • AstraZeneca’s Voydeya (danicopan) for treating extravascular hemolysis with paroxysmal nocturnal hemoglobinuria, a rare blood disease;
  • Bristol-Myers Squibb’s Cobenfy (xanomeline and trospium chloride) for treating schizophrenia;
  • GE HealthCare’s  Flyrcado (flurpiridaz F 18), a radioactive diagnostic drug to evaluate for myocardial ischemia and infarction;
  • Gilead Sciences’ Livdelzi (seladelpar) for treating primary biliary cholangitis;
  • Johnson & Johnson’s Lazcluze (lazertinib) for treating non-small cell lung cancer; and
  • Roche’s Itovebi (inavolisib) for treating locally advanced or metastatic breast cancer;

Small-molecules by far dominated new approvals from mid-sized to small companies, with many of the drugs indicated for rare diseases. Below is a roundup of small-molecule drug approvals from mid-sized and small companies by FDA’s CDER in 2024.

  • Akebia Therapeutics’ Vafseo (vadadustat) for treating anemia due to chronic kidney disease;
  • Allecra Therapeutics’ Exblifep (cefepime, enmetazobactam) for treating complicated urinary tract infections;
  • AOP Orphan Pharmaceuticals’ Rapiblyk (landiolol) for treating supraventricular tachycardia;
  • Ascendis Pharma’s Yorvipath (palopegteriparatide) for treating hypoparathyroidism, a rare disease where the parathyroid glands behind the thyroid do not produce enough parathyroid hormone;
  • Basilea Pharmaceutica’s Zevtera (ceftobiprole medocaril sodium for injection), an antibiotic for treating certain bloodstream infections, bacterial skin and associated tissue infections, and community-acquired bacterial pneumonia;
  • Botanix’s  Sofdra (sofpironium) for treating primary axillary hyperhidrosis, a chronic disorder characterized by uncontrollable excessive sweating without a recognizable cause;
  • Bracco’s Iomervu (iomeprol), a radiographic contrast agent;
  • BridgeBio Pharma’s Attruby (acoramidis) for treating cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis;
  • Day One Biopharmaceuticals’ Ojemda (tovorafenib) for treating relapsed or refractory pediatric low-grade glioma (i.e., brain tumor);
  • Geron’s Rytelo (imetelstat) for treating low- to intermediate-1 risk myelodysplastic syndromes, a group of progressive bone marrow disorders;
  • Idorsia’s Tryvio (aprocitentan) for treating hypertension;
  • IntraBio’s Aqneursa (levacetylleucine) for treating Niemann-Pick disease type C, a rare genetic disease resulting in progressive neurological symptoms and organ dysfunction;
  • Ionis Pharmaceuticals’Tryngolza (olezarsen) for treating familial chylomicronemia syndrome;
  • Ispen’s Iqirvo (elafibranor) for treating  primary biliary cholangitis in combination with ursodeoxycholic acid;
  • Iterum Therapeutics’ Orlynvah (sulopenem etzadroxil, probenecid) for treating uncomplicated urinary tract infections;
  • ITF Therapeutics’ (Italfarmaco) Duvyzat (givinostat) for treating Duchenne muscular dystrophy, a severe, inherited disease that causes progressive muscle weakness and degeneration;
  • LNHC’s Zelsuvmi (berdazimer) for treating molluscum contagiosum, a skin infection;
  • Lumicell’s Lumisight (pegulicianine), an optical imaging agent for detecting cancerous tissue;
  • Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) for treating noncirrhotic non-alcoholic steatohepatitis (NASH), a nonalcoholic fatty liver disease where liver inflammation, over time, can lead to liver scarring and liver dysfunction;
  • Neurocrine Biosciences’ Crenessity (crinecerfont) for treating classic congenital adrenal hyperplasia;
  • Servier’s Voranigo (vorasidenib) for treating Grade 2 astrocytoma or oligodendroglioma, two types of low-grade brain tumors;
  • Sun Pharma’s Leqselvi (deuruxolitinib) for treating severe alopecia areata, a common autoimmune disease, causing sudden hair loss;
  • Syndax Pharmaceuticals’ Revuforj (revumenib) for treating relapsed or refractory acute leukemia;
  • Verona Pharma’s Ohtuvayre (ensifentrine) for treating chronic obstructive pulmonary disease;
  • Vertex Pharmaceuticals’ Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) for treating cystic fibrosis;
  • X4 Pharmaceuticals’ Xolremdi (mavorixafor) for treating WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis);
  • Xcovery Holdings’ Ensacove (ensartinib) for treating non-small cell lung cancer; and
  • Zerva Therapeutics’ Miplyffa (arimoclomol), for treating Niemann-Pick disease type C

Biological approvals in 2024
In 2024, FDA’s CDER approved 16 biological therapeutics, which represented 32% of new drug approvals (new molecular entities and biological therapeutics) by FDA’s CDER. This was on par with 2023, when FDA’s CDER approved 17 new biological therapeutics, which represented 31% of new drug approvals by FDA’s CDER in 2023. FDA’s CDER reviews and approves new molecular entities and new biological therapeutics. Other biologic-based products, including blood products, vaccines, allergenics, tissues, and cellular and gene therapies, are reviewed and approved by a separate center within FDA, the Center for Biologics Evaluation and Research (CBER).

Biologics: large versus smaller companies
The 16 biologics approved by FDA’s CDER in 2024 were evenly divided between the large bio/pharmaceutical companies and small/mid-sized companies, with eight approvals from each group. Lilly led with two approvals, with Amgen, Astellas, Merck & Co., Novo Nordisk, Pfizer, and Roche each recording one biological approval by FDA’s CDER.

Biologics approved by FDA’s CDER from the large pharma companies were: Amgen’s Imdelltra (tarlatamab-dlle) for treating extensive stage small-cell lung cancer; Astellas’s Vyloy (zolbetuximab-clzb) in combination with fluoropyrimidine- and platinum-containing chemotherapy as a first-line treatment of adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction adenocarcinoma; Eli Lilly and Company’s Ebglyss (lebrikizumab-lbkz) for treating moderate-to-severe eczema (atopic dermatitis); Lilly’s Kisunla (donanemab-azbt) for treating early symptomatic Alzheimer’s disease; Merck & Co.’s Winrevair (sotatercept-csrk) for treating pulmonary arterial hypertension (PAH), one form of a broader condition known as pulmonary hypertension, which refers to high blood pressure in the lung; Novo Nordisk’s Alhemo (concizumab-mtci) for preventing or reducing the frequency of bleeding episodes in patients with hemophilia A or B; Pfizer’s Hympavzi (marstacimab-hncq) also for preventing or reducing bleeding episodes related to hemophilia A or B; and Roche’s Piasky (crovalimab-akkz) for treating paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease that causes red blood cells to break down.

Smal;-to-midsized companies accounted for the remaining biological approvals by FDA’s CDER in 2024 with eight companies recording one biological approval: BeiGene, Checkpoint Therapeutics, Galderman Laboatories,Hugel, ImmunityBio, Incyte, Jazz Pharmaceuticals, and Merus.   

Biologics approved in 2024 by FDA’s CDER from small to mid-sized companies included: BeiGene’s Tevimbra (tislelizumab-jsgr) for treating esophageal squamous cell carcinoma; Checkpoint Therapeutics’ Unloxcyt (cosibelimab-ipdl) for treating metastatic cutaneous squamous cell carcinoma, a  type of skin cancer that develops in the squamous cells of the epidermis; Galderman Labs’ Nemluvio (nemolizumab-ilto) for treating prurigo nodularis, a chronic skin disorder characterized by multiple pruritic papules, plaques, and nodules; Hugel’s Letybo (etibotulinumtoxinA-wlbg) for treating moderate-to-severe glabellar (frown) lines;  ImmunityBio’s Anktiva (nogapendekin alfa inbakicept-pmln) for treating a certain form of bladder cancer; Incyte’s Niktimvo (axatilimab-csfr) for treating chronic graft-versus-host disease after failure of at least two prior lines of systemic therapy; Jazz Pharmaceuticals’ Ziihera (zanidatamab-hrii) for treating previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, a rare and aggressive type of cancer that affects the organs of the biliary system, which includes the gallbladder and bile ducts; and Merus’ Bizengri (zenocutuzumab-zbco) for treating unresectable or metastatic non-small cell lung cancer.

Table 1: Approvals of New Molecular Entities and Biological Therapeutics by the Center for Drug Evaluation and Research, US Food and Drug Administration, 2024.

CompanyDrug: Brand name (active pharmaceutical ingredient), NDA or BLAIndication
Akebia TherapeuticsVafseo (vadadustat)
NDA
Anemia due to chronic kidney disease
Allecra TherapeuticsExblifep (cefepime & enmetazobactam)
NDA
Complicated urinary tract infections;
AmgenImdelltra (tarlatamab-dlle) BLAExtensive-stage small cell lung cancer
AOP Orphan
Pharmaceuticals
Rapiblyk (landiolol)
NDA
Supraventricular tachycardia
Ascendis PharmaYorvipath (palopegteriparatide)
NDA
Hypoparathyroidism
AstellasVyloy (zolbetuximab-clzb) BLAGastric or gastroesophageal junction adenocarcinoma, a rare cancer of the esophagus
AstraZenecaVoydeya (danicopan)
NDA
Extravascular hemolysis with paroxysmal nocturnal hemoglobinuria, a rare blood disease
Basilea
Pharmaceutica
Zevtera (ceftobiprole medocaril sodium)
NDA
Certain bloodstream infections, bacterial skin and associated tissue infections, and community-acquired bacterial pneumonia;
BeiGeneTevimbra (tislelizumab-jsgr) BLAUnresectable or metastatic esophageal cancer
BotanixSofdra (sofpironium)
NDA
Primary axillary hyperhidrosis (uncontrollable excessive sweating)
BraccoIomervu (iomeprol)
NDA
Radiographic contrast agent
BridgeBio PharmaAttruby (acoramidis)
NDA
Cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis
Bristol-Myers SquibbCobenfy (xanomeline and trospium chloride)
NDA
Schizophrenia;
Checkpoint TherapeuticsUnloxcyt (cosibelimab-ipdl) BLACutaneous squamous cell carcinoma
Day One BiopharmaceuticalsOjemda (tovorafenib)
NDA
Relapsed or refractory pediatric low-grade glioma (i.e., brain tumor)
Eli Lilly and CompanyEbglyss (lebrikizumab-lbkz) BLAModerate-to-severe atopic dermatitis (eczema)
Eli Lilly and CompanyKisunla (donanemab-azbt) BLAEarly symptomatic Alzheimer’s disease
GaldermaNemluvio
(nemolizumab-ilto)
BLA
Prurigo nodularis, a chronic skin disorder with itchy nodules
GE HealthCareFlyrcado (flurpiridaz F 18) NDARadioactive diagnostic drug to evaluate for myocardial ischemia and infarction.
GeronRytelo (imetelstat)
NDA
Low- to-intermediate risk myelodysplastic syndromes, a type of bone marrow disorders.
Gilead SciencesLivdelzi (seladelpar)
NDA
Primary biliary cholangitis
Hugel-AestheticsLetybo
(letibotulinumtoxinA-wlbg)
BLA
Temporarily improving the appearance of moderate-to-severe glabellar lines (i.e., frown lines
 IdorsiaTryvio (aprocitentan)
NDA
Hypertension
ImmunityBio
Anktiva (nogapendekin alfa
inbakicept-pmln)
Bladder cancer
IncyteNiktimvo (axatilimab-csfr) BLAChronic graft-versus-host disease
IntraBioAqneursa (levacetylleucine)
NDA
Niemann-Pick disease type C, a rare genetic disease resulting in progressive neurological symptoms and organ dysfunction
Ionis PharmaceuticalsTryngolza (olezarsen)
NDA
Familial chylomicronemia syndrome
IspenIqirvo (elafibranor)
NDA
Primary biliary cholangitis in combination with ursodeoxycholic acid
Iterum TherapeuticsOrlynvah (sulopenem etzadroxil and probenecid) NDAUncomplicated urinary tract infections
ITF Therapeutics Duvyzat (givinostat)
NDA
Duchenne muscular dystrophy
Jazz
Pharmaceuticals
Ziihera (zanidatamab-hrii) BLAUnresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer
Johnson & JohnsonLazcluze (lazertinib)
NDA
Non-small cell lung cancer
LNHCZelsuvmi (berdazimer)
NDA
Molluscum contagiosum, a skin infection
Lumicell  Lumisight (pegulicianine) NDAOptical imaging agent for detecting cancerous tissue
Madrigal
Pharmaceuticals
Rezdiffra (resmetirom)
NDA
Noncirrhotic non-alcoholic steatohepatitis (NASH)
Merck & Co. Inc.Winrevair (sotatercept-csrk) BLAPulmonary arterial hypertension
MerusBizengri
(zenocutuzumab-zbco)
BLA
Non-small cell lung cancer and pancreatic adenocarcinoma
Neurocrine BiosciencesCrenessity (crinecerfont) NDAClassic congenital adrenal hyperplasia
Novo NordiskAlhemo (concizumab-mtci) BLARoutine prophylaxis to prevent bleeding episodes in hemophilia A and B
PfizerHympavzi
(marstacimab-hncq)
BLA
Preventing or reducing bleeding episodes related to hemophilia A or B
RocheItovebi (inavolisib)
NDA
Locally advanced or metastatic breast cancer
RochePiasky (crovalimab-akkz)
BLA
Paroxysmal nocturnal hemoglobinuria, a rare blood disorder
ServierVoranigo (vorasidenib)
NDA
Grade 2 astrocytoma or olodendroglioma, two types of low-grade brain tumors
Sun PharmaLeqselvi (deuruxolitinib)
NDA
Severe alopecia areata (sudden hair loss)
Syndax
Pharmaceuticals
Revuforj (revumenib)
NDA
Relapsed or refractory acute leukemia
Verona PharmaOhtuvayre (ensifentrine) NDAChronic obstructive pulmonary disease
Vertex
Pharmaceuticals
Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor) NDACystic fibrosis
X4
Pharmaceuticals
Xolremdi (mavorixafor)
NDA
WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis)
Xcovery HoldingsEnsacove (ensartinib)
NDA
To treat non-small cell lung cancer
Zerva TherapeuticsMiplyffa (arimoclomol)
NDA
Niemann-Pick disease type C

Note: NDA is new drug application; BLA is biologics license application.

Source: Center for Drug Evaluation and Research, US Food and Drug Administration

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