Week's news analysis

2020 FDA approvals : close to a new record

By The Editor - 07 January 2021

As Commissioner Stephen Hahn will depart the FDA by January 20, when the Biden administration takes office, review of drug approvals in 2020 shows the Covid-19 pandemic did not slow the trend. According to the FDA, the Center for Drug Evaluation and Research approved 53 "first-of-their-kind medicines" for the US market. A better result than in 2019 (48 new molecular entities, NMEs), close to the 2018 record, when the CDER cleared 59 novel therapies.

This result does not contain vaccines, allergenic products, blood products, plasma derivatives, cellular and gene therapy products, which were approved in 2020 by the Center for Biologics Evaluation and Research. Nor does it count the emergency use authorizations (EUA) for two Covid-19 vaccines (from Pfizer-BioNtech and Moderna), issued by the FDA in December, or the EUA granted to two monoclonal antibody treatments (from Eli Lilly and Regeneron Pharmaceuticals Inc). Still in the period under review, the FDA authorized more than 300 Covid-19 test and collection kits, including 235 molecular diagnostics, 63 antibody tests and 11 antigen tests. "Our Center for Devices and Radiological Health (CDRH) has made enormous contributions to the Covid-19 response, with tests that can be used in laboratories, in doctors' offices and other point-of-care settings, and at home," Stephen Hahn explained in a news release. With respect to Covid-19 treatments, Gilead Sciences'antiviral Verlurky (remdesivir) was the first (and only) drug for treating the pandemic that received full approval by the FDA. The drug was approved in October for treating patients requiring hospitalization, 12 years of age and older and weighing at least 40 kilograms. Gilead had earlier received emergency use authorization by the FDA in May.



Nearly 60 %, or 30, of the novel drugs approved by the FDA in 2020 were for rare diseases. Among those Evrysdi from Genentech Inc (Roche Holdings), becoming the first oral treatment in the US for spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Also approved in November 2020, Eiger BioPharmaceuticals Inc.'s Zokinvy (lonafarnib), the first drug in the US to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and to treat certain processing-deficient progeroid laminopathies in patients as young as age 1. Progeria is a rare genetic disorder that causes children to rapidly age. In August, the FDA's CDER also approved Viltepso, a treatment for Duchenne muscular dystrophy (DMD), from Nippon Shinyaku Co. Ltd.The rare, debilitating, progressive and ultimately fatal childhood genetic disease is caused by mutations in the gene encoding dystrophin, a protein that plays a key role in maintaining muscle integrity.

The FDA continued to push through approvals on a large number of oncology therapies. In October, it fully approved Venclexta (venetoclax) in combination with Vidaza (azacytidine), Dacogen (decitabine) or low dose cytarabine (LDAC) in newly diagnosed patients with Acute Myeloid Leukemia (AML) aged 75 years or older. In May, the FDA approved Qinlock (ripretinib), to treat patients with advanced gastrointestinal stromal tumor (GIST). In May, the FDA approved Retevmo (selpercatinib), the first of its kind, to treat non-small cell lung cancer (NSCLC), medullary thyroid cancer and other types of thyroid cancers with RET alterations. In April, the FDA's approval of Trodelvy, the first antibody-drug conjugate to treat patients with pretreated metastatic triple-negative breast cancer was big news for patients

In 2020, CDER also approved two novel antibody treatments for Ebola : Regeneron's Inmazeb in October and Ridgeback Biotherapeutics LP's Ebanga in December.

Last but not least, 2020 was a poor year for biosimilars, with only three approved therapies (the fewest since 2016): Nyvepria (pegfilgrastim), a biosimilar version of Amgen Inc.'s Neulasta, developed by Pfizer. It is a leukocyte growth factor used to treat the depletion of white blood cells caused by cancer. In July, Viatris was granted to sell Hulio, a biosimilar of AbbVie Inc.'s injectable arthritis drug Humira (adalimumab), while in December, Amgen won the FDA's approval to market Riabni, a biosimilar version of Rituxan (rituximab), a cancer medicine comarketed by Biogen and Genentech.

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