The Small Molecule and Biologic Pharmacopoeia Continues to Grow
This cohort is nearly 50% bigger than the new approval class of 2022 (see Figure 1).
Figure 1: 30 years of novel FDA approvals. Annual numbers of new molecular entities (NMEs) and biologics license applications (BLAs) approved by the FDA’s Center for Drug Evaluation and Research (CDER). See Table 1 for new approvals in 2023. Products approved by the Center for Biologics Evaluation and Research (CBER), including vaccines and gene therapies, are not included in this drug count. Source: FDA & Nature.
The Nature article, 2023 FDA approvals, summarizes in more details the CBER approvals that includes vaccine and gene therapies.
By therapeutic area, oncology continues to accumulate the most approvals. CDER gave a green light to 13 (24%) new cancer therapies in 2023. Neurology came in second, with 9 (16%) approvals, also in line with recent trends. Infectious diseases and haematology tied for third, with 5 (9%) apiece.
| Drug Name | Active Ingredient | Approval Date | FDA-approved use on approval date* |
|---|---|---|---|
| Leqembi | lecanemab-irmb | 1/6/2023 | To treat Alzheimer’s disease Press Release |
| Brenzavvy | bexagliflozin | 1/20/2023 | To improve glycemic control in adults with type 2 diabetes mellitus as an adjunct to diet and exercise |
| Jaypirca | pirtobrutinib | 1/27/2023 | To treat relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor |
| Orserdu | elacestrant | 1/27/2023 | To treat estrogen receptor-positive, human epidermal growth factor receptor 2-negative, ESR1-mutated, advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy |
| Jesduvroq | daprodustat | 2/1/2023 | To treat anemia caused by chronic kidney disease for adults on dialysis for at least four months Press Release |
| Lamzede | velmanase alfa-tycv | 2/16/2023 | To treat non-central nervous system manifestations of alpha-mannosidosis |
| Filspari | sparsentan | 2/17/2023 | To reduce proteinuria in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression |
| Skyclarys | omaveloxolone | 2/28/2023 | To treat Friedrich’s ataxia |
| Zavzpret | zavegepant | 3/9/2023 | To treat migraine |
| Daybue | trofinetide | 3/10/2023 | To treat Rett syndrome |
| Zynyz | retifanlimab-dlwr | 3/22/2023 | To treat metastatic or recurrent locally advanced Merkel cell carcinoma |
| Rezzayo | rezafungin | 3/22/2023 | To treat candidemia and invasive candidiasis |
| Joenja | leniolisib | 3/24/2023 | To treat activated phosphoinositide 3-kinase delta syndrome |
| Qalsody | tofersen | 4/25/2023 | To treat amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation |
| Elfabrio | pegunigalsidase alfa-iwxj | 5/9/2023 | To treat confirmed Fabry disease |
| Veozah | fezolinetant | 5/12/2023 | To treat moderate to severe hot flashes caused by menopause Press Release |
| Miebo | perfluorhexyloctane | 5/18/2023 | To treat signs and symptoms of dry eye disease |
| Epkinly | epcoritamab-bysp | 5/19/2023 | To treat relapsed or refractory diffuse large B-cell lymphoma (not otherwise specified) and high-grade B-cell lymphoma after two or more lines of systemic therapy |
| Xacduro | sulbactam, durlobactam | 5/23/2023 | To treat hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex Press Release |
| Paxlovid | nirmatrelvir, ritonavir | 5/25/2023 | To treat mild-to-moderate COVID-19 in adults at high risk for progression to severe COVID-19 Press Release |
| Posluma | flotufolastat F 18 | 5/25/2023 | To use with positron emission tomography imaging in certain patients with prostate cancer |
| Inpefa | sotagliflozin | 5/26/2023 | To treat heart failure |
| Columvi | glofitamab-gxbm | 6/15/2023 | To treat diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy |
| Litfulo | ritlecitinib | 6/23/2023 | To treat severely patchy hair loss |
| Rystiggo | rozanolixizumab-noli | 6/26/2023 | To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive |
| Ngenla | somatrogon-ghla | 6/27/2023 | To treat growth failure due to inadequate secretion of endogenous growth hormone |
| Beyfortus | nirsevimab-alip | 7/17/2023 | To prevent respiratory syncytial virus (RSV) lower respiratory tract disease Press Release |
| Vanflyta | quizartinib | 7/20/2023 | To use as part of a treatment regimen for newly diagnosed acute myeloid leukemia that meets certain criteria |
| Xdemvy | lotilaner | 7/25/2023 | To treat Demodex blepharitis |
| Zurzuvae | zuranolone | 8/4/2023 | To treat postpartum depression Press Release |
| Izervay | avacincaptad pegol | 8/4/2023 | To treat geographic atrophy secondary to age-related macular degeneration |
| Talvey | talquetamab-tgvs | 8/9/2023 | To treat adults with relapsed or refractory multiple myeloma who have received at least four prior therapies |
| Elrexfio | elranatamab-bcmm | 8/14/2023 | To treat adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy |
| Sohonos | palovarotene | 8/16/2023 | To reduce the volume of new heterotopic ossification in adults and pediatric patients (aged 8 years and older for females and 10 years and older for males) with fibrodysplasia ossificans progressiva |
| Veopoz | pozelimab-bbfg | 8/18/2023 | To treat patients 1 year old and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease |
| Aphexda | motixafortide | 9/8/2023 | To use with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma |
| Ojjaara | momelotinib | 9/15/2023 | To treat intermediate or high-risk myelofibrosis in adults with anemia |
| Exxua | gepirone | 9/22/2023 | To treat major depressive disorder |
| Pombiliti | cipaglucosidase alfa-atga | 9/28/2023 | To treat late-onset Pompe disease |
| Rivfloza | nedosiran | 9/29/2023 | To lower urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function |
| Velsipity | etrasimod | 10/12/2023 | To treat moderately to severely active ulcerative colitis in adults |
| Zilbrysq | zilucoplan | 10/17/2023 | To treat generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive |
| Bimzelx | bimekizumab | 10/17/2023 | To treat moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy |
| Agamree | vamorolone | 10/26/2023 | To treat Duchenne muscular dystrophy |
| Omvoh | mirikizumab-mrkz | 10/26/2023 | To treat ulcerative colitis |
| Loqtorzi | toripalimab-tpzi | 10/27/2023 | To treat recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies |
| Fruzaqla | fruquintinib | 11/8/2023 | To treat refractory, metastatic colorectal cancer |
| Defencath | taurolidine, heparin | 11/15/2023 | To reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis through a central venous catheter |
| Augtyro | repotrectinib | 11/15/2023 | To treat ROS1-positive non-small cell lung cancer |
| Ryzneuta | efbemalenograstim alfa-vuxw | 11/16/2023 | To treat neutropenia |
| Truqap | capivasertib | 11/16/2023 | To treat breast cancer that meets certain disease criteria |
| Ogsiveo | nirogacestat | 11/27/2023 | To treat adults with progressing desmoid tumors who require systemic treatment |
| Fabhalta | iptacopan | 12/5/2023 | To treat paroxysmal nocturnal hemoglobinuria |
| Filsuvez | birch triterpenes | 12/18/2023 | To treat wounds associated with dystrophic and junctional epidermolysis bullosa |
| Wainua | eplontersen | 12/21/2023 | To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis |
By modality, the medical toolbox continues to get more diverse. 2023 saw an increase in the number of nucleotide-based therapeutics, including a second RNA-aptamer product (see Figure 2).
Figure 2: CDER approvals by modality. Small molecules, including peptides of up to 40 amino acids in length, and oligonucleotides are approved as new molecular entities (NMEs). Protein-based candidates are approved through biologics license applications (BLAs). mAb, monoclonal antibody; siRNA, small interfering RNA. Source: Nature Reviews Drug Discovery.
Source: FDA & Nature Journal.
