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Pandemic not a barrier to US 2020 drug approvals; FDA clears 53 novel medicines

The U.S. Food and Drug Administration's Center for Drug Evaluation and Research approved 53 first-of-their-kind medicines in 2020, demonstrating the pandemic did not slow the agency's efforts in clearing novel drugs for the U.S. market.

The FDA tied its 1996 record for approving new molecular entities, or NMEs — an all-time high that was shattered in 2018 when regulators in the Center for Drug Evaluation and Research, or CDER, cleared 59 novel therapies.

CDER approved 48 NMEs in 2019.

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US FDA Commissioner Stephen Hahn
Source: U.S. Food and Drug Administration

The 53 NME approvals in 2020 will give FDA Commissioner Stephen Hahn a high note on which to depart the agency after a year of defending his actions in the pandemic against a number of critics, including President Donald Trump, who made unsubstantiated claims the regulatory chief and the scientists he oversees moved too slow in authorizing a COVID-19 vaccine to thwart the Republican's failed effort to keep control of the White House for four more years.

Like other political appointees, Hahn is expected to resign by Jan. 20, when the Biden administration takes office. Biden has yet to disclose his choice to lead the FDA.

Among the 53 NMEs approved by CDER in 2020 was Gilead Sciences Inc.'s COVID-19 drug Veklury, also known as remdesivir. The marketed indication for COVID-19 cleared Oct. 22, 2020, for people as young as age 12 requiring hospitalization was not as broad as the FDA had granted under its May 1, 2020, emergency use authorization, or EUA.

Veklury remains the only FDA-approved treatment for COVID-19, though the agency has granted EUAs to two monoclonal antibody treatments from Eli Lilly and Co. and Regeneron Pharmaceuticals Inc. Hahn's Aug. 23, 2020, decision to authorize convalescent plasma for emergency use was widely criticized by a number of scientists and public health experts, who questioned the data on which the EUA was granted.

In 2020, CDER also approved two novel antibody treatments for Ebola — another highly infectious disease that crippled Guinea, Liberia and Sierra Leone during the 2013 to 2016 West Africa epidemic and most recently devastated the Democratic Republic of the Congo, which has experienced nearly a dozen outbreaks of the disease. That country's latest Ebola outbreak was declared over in November 2020.

On Oct. 14, 2020, regulators cleared Regeneron's Inmazeb — the first FDA-approved Ebola treatment — and on Dec. 21, 2020, the agency approved Ridgeback Biotherapeutics LP's Ebanga as the second therapy in the U.S. for the disease.

There remains only one Ebola vaccine licensed by the FDA — Merck & Co. Inc.'s Ervebo, which the regulator cleared on Dec. 19, 2019, days after Hahn took office.

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Rare disease drugs

Nearly 60%, or 30, of the novel drugs approved by the FDA in 2020 are for rare diseases.

Among those was Eiger BioPharmaceuticals Inc.'s Zokinvy, or lonafarnib, the first drug in the U.S. 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. People with the disease often die before they reach their mid-teens.

U.S. National Institutes of Health Director Francis Collins noted in a Nov. 23, 2020, tweet that his lab at the agency discovered the gene that causes progeria and did much of the preclinical work that led to the development of Zokinvy.

The FDA's CDER also approved Viltepso, a treatment for Duchenne muscular dystrophy, or DMD, from Nippon Shinyaku Co. Ltd. unit NS Pharma Inc. 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.

Viltepso was the second drug approved in the U.S. for patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

The first DMD drug granted approval in the U.S. was Sarepta Therapeutics Inc.'s Vyondys 53, which the FDA cleared for the market in December 2019 after initially rejecting it.

Both Viltepso and Vyondys 53 must complete successful trials confirming their benefit to remain on the U.S. market.

Evrysdi from Roche Holding AG unit Genentech Inc. was another rare disease drug approved in 2020 — becoming the first oral treatment in the U.S. for spinal muscular atrophy, or SMA, a rare and often fatal genetic disease affecting muscle strength and movement. SMA affects mostly infants and children.

In 2016, the FDA approved Biogen Inc.'s SMA treatment Spinraza, which must be administered intrathecally. The FDA's Center for Biologics Evaluation and Research licensed Novartis AG's $2.1 million SMA gene therapy Zolgensma in May 2019.

First-time generics

As of Dec. 14, 2020, the FDA had approved 65 first-time generic medicines — the first copycats of brand-name small-molecule pills allowed to be marketed in the U.S. However, the 2020 total for first time generics was the lowest in the past five years.

Included in those 2020 generic approvals was the first lower-cost version of Daraprim — a nearly 70-year-old parasitic infection medicine whose price had shot up from $13.50 a pill to $750 per tablet, or about 5,000%, in August 2015.

The Daraprim price hike by Turing Pharmaceuticals LLC, which changed its name to Vyera Pharmaceuticals after its CEO Martin Shkreli left the company, was at the center of a number of investigations opened by Congress and the Federal Trade Commission.

The first generic approval of Daraprim was awarded to Cerovene Inc. in February 2020.

In August 2020, Viatris Inc., formerly Mylan NV, also won a first generic approval for its version of Biogen's moneymaker treatment Tecfidera, which is used to treat multiple sclerosis.

Biogen had reported $4.4 billion in 2019 sales for Tecfidera, or about 40% of the company's revenue.

Biosimilars

The FDA only approved three lower-cost versions of biologic therapies, or biosimilars, in 2020 — the fewest since 2016.

In June 2020, the FDA told Pfizer Inc. it could market Nyvepria, a biosimilar version of Amgen Inc.'s Neulasta, also known as pegfilgrastim, a leukocyte growth factor used to treat the depletion of white blood cells caused by cancer.

Viatris was also granted the go-ahead in July 2020 in the U.S. to sell Hulio, a biosimiar of AbbVie Inc.'s injectable arthritis drug Humira, or adalimumab — the top-selling medicine in the world in 2019, bringing in about $15 billion for the company.

In December 2020, Amgen won the FDA's approval to market Riabni, a biosimilar version of Rituxan, or rituximab, a cancer medicine comarketed in the U.S. by Biogen and Genentech.

The FDA had already previously cleared biosimilars of all three of the brand-name biologics.

Congress gave the FDA the authority to approve biosimilars under the Biologics Price Competition and Innovation Act, which was adopted in 2010 as part of the Affordable Care Act. There have been 29 biosimilars approved in the U.S. since 2015, the first year the FDA cleared one of those products for the U.S. market.