FDA/CDER Approvals in 2023: A Comprehensive Overview of Novel Drugs

In 2023, the FDA’s Center for Drug Evaluation and Research (CDER) approved a total of 55 innovative drugs as of December 26. This represents a remarkable increase of approximately 51% compared to the previous year, 2022.

Among the pharmaceutical companies, Pfizer leads with the highest number of approvals, with 6 newly approved drugs, accounting for 11% of the total approvals. These drugs cover a range of conditions, including migraine, COVID-19, alopecia areata, pediatric growth hormone deficiency, relapsed/refractory multiple myeloma, and ulcerative colitis.

The list of approved drugs:

The Range of Therapeutic Approvals in 2023: Biogen, AstraZeneca, Chiesi, and UCB

Biogen follows with 4 approvals (7%), including drugs for Alzheimer’s disease, Friedreich’s ataxia, ALS, and postpartum depression. AstraZeneca and Chiesi both have 3 approvals each (5%), with AstraZeneca focusing on conditions like RSV, breast cancer, and hereditary transthyretin-mediated amyloidosis, while Chiesi addresses alpha-mannosidosis, Fabry disease, and epidermolysis bullosa. UCB also holds 3 approvals (5%), including treatments for myasthenia gravis and plaque psoriasis.

The newly approved drugs belong to various therapeutic classes, with small molecules accounting for the majority at 55%. Therapeutic monoclonal antibodies (mAbs) represent 22% of approvals, including different types of IgG antibodies. Peptides and proteins make up 14% of the approvals, including synthetic peptides, recombinant enzymes/hormones, and fusion proteins. DNA/RNA-based drugs account for 7% of approvals, and there is one natural extract comprising 2% of the total.

2023 FDA/CDER Drug Approvals: Therapeutic Areas and Regulatory Pathways

In terms of therapeutic areas, oncology leads with 31% of approvals, covering a wide range of cancer types. Neurology and autoimmune disorders follow closely with 11% each, addressing conditions such as Alzheimer’s, ALS, migraine, and ulcerative colitis. Infectious diseases and metabolic disorders each have 9% of approvals, while ophthalmology rounds out the list with 5%.

Additionally, the regulatory pathway/designations for these drugs vary, with 9 receiving accelerated approval, 18 designated as orphan drugs, 16 granted fast track designation, 24 undergoing priority review, and 9 recognized as breakthrough therapies.

FDA’s Remarkable Year in Drug Innovation: 2023’s Novel Drug Approvals

In a year marked by groundbreaking achievements in the field of pharmaceuticals, the FDA (Food and Drug Administration) has once again proven its commitment to advancing healthcare. In 2023, the FDA approved a remarkable total of 55 novel drugs. These groundbreaking medications, categorized as either new molecular entities (NMEs) introduced through New Drug Applications (NDAs) or revolutionary therapeutic biologics brought forth via Biologics License Applications (BLAs), signify a significant leap forward in the world of medicine.

What sets these novel drugs apart is that their active ingredients had not previously received approval for use within the United States. This speaks volumes about the dedication of both pharmaceutical companies and regulatory bodies to delivering innovative solutions to address unmet medical needs.

First-In-Class Innovations

In 2023, CDER highlighted 36% of newly approved drugs as ‘first-in-class,’ introducing innovative mechanisms distinct from existing treatments. Notable approvals included:

• Daybue (trofinetide) oral solution: Pioneering treatment for Rett syndrome, a rare genetic neurological disorder in patients aged two and older
• Jesduvroq (daprodustat) tablets: Groundbreaking oral solution for anemia in adults with chronic kidney disease undergoing dialysis for over four months
• Miebo (perfluorohexyloctane) ophthalmic solution: Innovative approach to relieving dry eye disease, addressing inflammation and eye lubrication
• Paxlovid (nirmatrelvir and ritonavir, co-packaged) tablets: First oral antiviral pill for mild-to-moderate COVID-19 in high-risk adults
• Skyclarys (omaveloxolone) capsules: Revolutionary therapy for Friedreich’s ataxia, a rare inherited disease causing nervous system damage
• Talvey (talquetamab-tgvs) injection: Offering hope to adults with refractory or relapsed multiple myeloma
• Veozah (fezolinetant) tablets: Addressing vasomotor symptoms associated with menopause
• Xdemvy (lotilaner) ophthalmic solution: Innovative solution for Demodex blepharitis, chronic eyelid inflammation

Advancements in Rare Disease Treatments

In 2023, CDER granted orphan drug designation to 51% of its 55 novel drug approvals, catering to rare diseases affecting fewer than 200,000 individuals in the U.S. Notable examples include:

• Fabhalta (iptacopan) tablets: Oral treatment for paroxysmal nocturnal hemoglobinuria
• Filspari (sparsentan) tablets: Reducing proteinuria in adults with primary immunoglobulin A (IgA) nephropathy
• Jaypirca (pirtobrutinib) tablets: Relief for mantle cell lymphoma and chronic lymphocytic leukemia patients
• Joenja (leniolisib) tablets: First treatment for activated phosphoinositide 3-kinase delta syndrome (APDS) in patients 12 years and older
• Lamzede (velmanase alfa) injection: Alleviating non-central nervous system manifestations of alpha-mannosidosis

These approvals in 2023 reflect CDER’s commitment to addressing unmet patient needs, particularly in rare diseases. Stay tuned for more insights into the remarkable advancements of this exceptional year in pharmaceutical innovation.

Innovative Strategies: Streamlining Drug Development and Approval

In 2023, the FDA’s Center for Drug Evaluation and Research (CDER) adopted regulatory measures to streamline and enhance drug reviews. These approaches boosted flexibility, efficiency, and collaboration with drug developers, resulting in shorter review times while maintaining rigorous safety and effectiveness standards.

Fast Track Advancements: CDER granted Fast Track status to 45% of 55 novel drugs in 2023, expediting their development and review. This designation encouraged FDA-developer communication, rolling application reviews, and included Agamree, Beyfortus, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Filsuvez, Fruzaqla, Izervay, Jaypirca, Lamzede, Leqembi, Ogsiveo, Orserdu, Paxlovid, Qalsody, Rezzayo, Skyclarys, Truqap, Vanflyta, Veopoz, Xacduro, Zurzuvae, Zynyz.

Pioneering Breakthrough Therapy Designations: CDER labeled 16% of novel drugs as ‘Breakthrough Therapies,’ combining Fast Track benefits with intensive FDA guidance. Drugs like Elrexfio, Fabhalta, Izervay, Leqembi, Loqtorzi, Ogsiveo, Pombiliti, Rivfloza, and Talvey earned this status.

Priority Review for Significant Advances: A remarkable 56% of novel drugs received Priority Review, recognizing their potential to address severe conditions and offer substantial safety or effectiveness improvements. Drugs like Augtyro, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Epkinly, Filspari, Filsuvez, Fruzaqla, Izervay, Jaypirca, Joenja, Lamzede, Leqembi, Loqtorzi, Ogsiveo, Orserdu, Paxlovid, Qalsody, Rezzayo, Rystiggo, Skyclarys, Sohonos, Talvey, Truqap, Vanflyta, Veopoz, Xacduro, Zurzuvae, and Zynyz achieved this designation.

Accelerated Approvals for Critical Needs: CDER granted Accelerated Approval to 16% of novel drugs, addressing urgent medical needs with quicker timelines. These approvals often rely on surrogate clinical endpoints and mandate post-approval studies for safety confirmation. Drugs like Columvi, Elrexfio, Epkinly, Filspari, Jaypirca, Leqembi, Qalsody, Talvey, and Zynyz were approved this way.

Expeditious Drug Approvals

In 2023, 65% of novel drugs benefitted from expedited programs (e.g., Fast Track, Breakthrough Therapy, Priority Review, or Accelerated Approval). CDER’s commitment to swift innovation led to faster access to treatments for patients. Examples include Agamree, Augtyro, Beyfortus, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Epkinly, Fabhalta, Filspari, Filsuvez, Fruzaqla, Izervay, Jaypirca, Joenja, Lamzede, Leqembi, Loqtorzi, Ogsiveo, Orserdu, Paxlovid, Pombiliti, Qalsody, Rezzayo, Rivfloza, Rystiggo, Skyclarys, Sohonos, Talvey, Truqap, Vanflyta, Veopoz, Xacduro, Zurzuvae, and Zynyz.

Meeting PDUFA Goals

CDER met or exceeded PDUFA goal dates for 89% of 2023’s 55 novel drugs. This underscores the agency’s commitment to predictability and timely decisions. Drugs approved on or before their PDUFA goals included Agamree, Aphexda, Augtyro, Beyfortus, Brenzavvy, Columvi, Daybue, Defencath, Elfabrio, Elrexfio, Epkinly, Exxua, Fabhalta, Filspari, Filsuvez, Fruzaqla, Inpefa, Izervay, Jaypirca, Jesduvroq, Joenja, Lamzede, Leqembi, Litfulo, Miebo, Ogsiveo, Ojjaara, Omvoh, Orserdu, Paxlovid, Posluma, Qalsody, Rezzayo, Rivfloza, Skyclarys, Sohonos, Talvey, Truqap, Vanflyta, Velsipity, Veopoz, Veozah, Wainua, Xacduro, Xdemvy, Zavzpret, Zilbrysq, Zurzuvae, and Zynyz.

Efficient First-Cycle Approvals

A remarkable 84% of novel drugs in 2023 were approved on their ‘first cycle’ of review, showcasing CDER’s dedication to clear guidance. Examples include Agamree, Aphexda, Augtyro, Beyfortus, Brenzavvy, Columvi, Daybue, Elrexfio, Epkinly, Fabhalta, Filspari, Fruzaqla, Inpefa, Izervay, Jaypirca, Jesduvroq, Joenja, Lamzede, Leqembi, Litfulo, Miebo, Ogsiveo, Ojjaara, and Orserdu.

Expanding Horizons for Approved Drugs

Following initial approval, drugs can gain new uses. In 2023, CDER approved new uses for drugs like Ayvakit (treating indolent systemic mastocytosis) and Kevzara (for polymyalgia rheumatica).

Additional Approvals for Known Drugs

CDER approved new uses or indications for previously approved drugs in 2023:

• Ayvakit: Approved for indolent systemic mastocytosis
• Eylea: Approved for retinopathy of prematurity (ROP)
• Ilaris: Approved for gout flares
• Jemperli: Approved for endometrial cancer
• Kevzara: Approved for polymyalgia rheumatica
• Keytruda: Approved for various applications
• Linzess: Approved for pediatric functional constipation
• Lonsurf: Approved for metastatic colorectal cancer
• Lynparza: Approved for metastatic and castration-resistant prostate cancer
• Padcev: Approved for for advanced urothelial cancer
• Polivy: Approved for relapsed and untreated lymphoma
• Rexulti: Approved for dementia-related agitation
• Rinvoq: Approved for Crohn’s disease
• Talzenna (talazoparib) capsules, approved in 2018 for breast cancer, gained 2023 approval for prostate cancer in combination therapy.
• Tukysa: Approved for colorectal cancer.
• Verzenio: Approved for early breast cancer.

CDER also expanded drug use to pediatric patients under specific legislation, improving access for younger populations.

Biosimilar Approvals

In 2023, CDER approved five new biosimilars for various reference products, including interchangeable biosimilars. These biosimilars offer alternatives to FDA-approved biologics, potentially reducing treatment costs.

Here’s a summary of the 2023 biosimilar approvals:

• Avzivi (bevacizumab-tnjn) injection: Approved for colorectal cancer and hepatocellular carcinoma, referencing Avastin
• Tofidence (tocilizumab-bavi) injection: Approved for arthritis, including rheumatoid arthritis, referencing Actemra
• Tyruko (natalizumab-sztn) injection: Approved for multiple sclerosis and Crohn’s disease, referencing Tysabri
• Wezlana (ustekinumab-auub) injection: Approved for psoriasis, psoriatic arthritis, Crohn’s disease, and ulcerative colitis, referencing Stelara, with interchangeable status
• Yuflyma (adalimumab-aaty) injection: Approved for various inflammatory conditions, referencing Humira
• Abrilada (adalimumab-afzb) injection, initially approved in 2019, gained interchangeable status, referencing Humira
• Byoovis (ranibizumab-nuna) injection, initially approved in 2021, also received interchangeable status, referencing Lucentis
• Since 2015, CDER approved 45 biosimilars for 14 reference products, enhancing competition and potentially reducing costs

Since 2015, CDER has granted approval to 45 biosimilars for 14 different reference products. These include multiple biosimilars for some of the best-selling biologics in the U.S., such as Humira, Neulasta, Herceptin, Avastin, Remicade, Rituxan, Neupogen, Lantus, Enbrel, and Lucentis. Increased competition resulting from multiple biosimilars for a single reference product has the potential to reduce costs for both patients and the healthcare system.

Noteworthy Approvals in 2023

• Airsupra (albuterol and budesonide) inhalation aerosol: Approved for bronchoconstriction and asthma attack prevention in adults, combining albuterol and budesonide
• Akeega (niraparib and abiraterone acetate) tablets: Approved for specific prostate cancer alongside prednisone, combining niraparib and abiraterone
• Brixadi (buprenorphine) extended-release injection: Approved for opioid use disorder in various forms and doses
• Entyvio (vedolizumab) injection: Extended approval for under-the-skin injections in ulcerative colitis cases, allowing self-administration
• Hepzato (melphalan hydrochloride) injection: Approved for liver metastases in uveal melanoma patients
• Lampit (nifurtimox) tablets: Full approval for pediatric Chagas disease treatment
• Lodoco (colchicine) tablets: Approved to reduce cardiovascular risk
• Mydcombi (tropicamide and phenylephrine hydrochloride) ophthalmic spray: Approved for pupil dilation
• Narcan (naloxone hydrochloride) nasal spray: Gained nonprescription status for opioid overdose reversal
• Opill (norgestrel) tablets: The first nonprescription daily oral contraceptive
• Opvee (nalmefene hydrochloride) nasal spray: Approved for opioid overdose reversal in a new form
• Prevymis (letermovir) tablets and injection: Extended approval to include kidney transplant recipients for CMV prevention
• RiVive (naloxone hydrochloride) nasal spray: Approved for nonprescription use to reverse opioid overdose effects
• Ryzumi (phentolamine) ophthalmic solution: Approved for pharmacologically induced mydriasis
• Syfovre (pegcetacoplan) injection: Approved for intravitreal use to treat geographic atrophy from age-related macular degeneration (AMD)
• Technegas (technetium Tc 99m labeled carbon) inhalation aerosol: Approved as a diagnostic agent for lung ventilation and pulmonary embolism evaluation
• Zepbound (tirzepatide) injection: Approved for chronic weight management in adults with obesity or overweight and at least one weight-related comorbidity
• Lampit tablets: Transitioned from Accelerated Approval to full approval for Chagas disease treatment in 2023

Drug approvals involve rigorous evaluation, considering safety, efficacy, disease severity, available treatments, and patient populations. External experts, advocates, industry, academics, and community members contribute to informed regulatory decisions, recognizing the impact on public health. Patients should review drug labeling and consult healthcare professionals for suitable treatment options.

We extend our sincere appreciation to Daniel Neumeier for his valuable contributions to the industry in preparing this insightful analysis on FDA/CDER approvals for 2023.

Resource: FDA, 2024

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