US Leads in Drug Approvals in 2019

In 2019, the 48 new drugs approved by FDA’s Center for Drug Evaluation and Research (CDER) fell short of the record-breaking 59 approvals in 2018; the number of biologic-based drug approvals also was down slightly (1). Despite the drop, the number of approvals was significant, many first-in-class therapies were introduced, and more drugs were approved in 2019 for US patients compared with other regions around the world.

Skyrizi (risankizumab-rzaa) from AbbVie for treatment of psoriasis was the only treatment approved in the United States, Europe, and Japan in 2019, with approval in Japan approximately one month before the US and Europe. A number of biologic drugs approved by FDA in 2018 were approved by the European Medicines Agency (EMA) and/or Japan’s Pharmaceuticals and Medical Devices Agency in 2019, including Crysvita (burosumab), Trogarzo (ibalizumab), Ajovy (fremanezumab), Libtayo (cemiplimab), Talzenna (talazoparib tosylate), and Ultomiris (ravulizumab) (2).

Historically, the US has led in the number of drug approvals. One study reported that between 2011 and 2015, FDA approved 170 new therapeutic agents, while EMA granted marketing authorization to 144 drugs. FDA approved a higher percentage of orphan drugs than EMA (43.5% vs. 25.0% of the total) and the median total review time at FDA was, on average, 60 days shorter (3).

Another study found that while FDA and EMA have agreed on the majority of approval decisions, the agencies did reach different conclusions regarding efficacy, on the same data or differing data (4).

FDA’s Center for Biologics Evaluation and Research had several notable approvals in 2019. Zolgensma (onasemnogene abeparvovec-xioi), from Novartis, was the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy.

In addition, three vaccines were approved in 2019: Dengvaxia (Sanofi Pasteur) to prevent dengue disease in individuals nine through 16 years of age, Jynneos (Bavarian Nordic A/S) to prevent smallpox and monkey pox disease in adults, and Vaxelis (MCM Vaccine Company), to prevent diphtheria, tetanus, pertussis, poliomyelitis, hepatitis B, and invasive disease due to Haemophilus influenza type b, for use in children from six weeks through four years of age (5).

Biologic-based therapies approved by CDER in 2019 include:

• Enhertu (fam-trastuzumab deruxtecan-nxki) from Daiichi Sankyo for the treatment of adults with unresectable or metastatic HER2-positive breast cancer 

• Padcev (enfortumab vedotin-ejfv) from Astellas Pharma US for the treatment of urothelial cancers

• Reblozyl (luspatercept–aamt) from Celgene, which reduces the need for blood transfusions in anemic patients with beta thalassemia

• Cablivi (caplacizumab-yhdp) injection from Ablynx for the treatment of adult patients with acquired thrombotic thrombocytopenic purpura

• Adakveo (crizanlizumab-tmca) from Novartis for vaso-occlusive crisis, a complication of sickle cell disease

• Beovu (brolucizumab) from Novartis for the treatment of wet age-related macular degeneration

• Polivy (polatuzumab vedotin-piiq) from Genentech to treat adult patients with diffuse large B-cell lymphoma

• Evenity (romosozumab-aqqg) from Amgen to treat osteoporosis

• Jeuveau (prabotulinumtoxinA-xvfs) from EVOLUS for the temporary improvement in the appearance of moderate to severe glabellar lines.

References

1. FDA, New Drugs at FDA: CDER’s New Molecular Entities and New Therapeutic Biological Products, www.fda.gov, accessed Dec. 30, 2019.
2. Kyoto Encyclopedia of Genes and Genomes Drug Database, “New Drug Approvals in the USA, Europe and Japan,” www.genome.jp.com, accessed Dec. 18, 2019.
3. N. S. Downing, A. D. Zhang, J. S. Ross, N Engl J Med 2017; 376:1386-1387.
4. M. Kashoki, et. al., Clin. Pharmacol. Ther., July 15, 2019.
5. P. Marks, FY 2019 Report from the Director, www.fda.gov, Dec. 26, 2019.