MHE Publication, MHE December 2020, Volume 30, Issue 12
Pre-pandemic momentum helped keep the number of novel therapies approved by the FDA high, and then COVID-19 tested just how fast the pipeline could adjust and expand.
2020 drug approvals
Some notable 2020 FDA approvals of new drugs
Tepezza (teprotumumab-trbw). Approved in January, Tepezza is the first and only FDA-approved treatment for thyroid eye disease, a rare and progressive inflammatory disease that affects the eye and nearby orbital tissues that can result in eye protrusion and visual impairment.
Zeposia (ozanimod). Approved in March, Zeposia is a treatment for adults with relapsing forms of multiple sclerosis (MS), including relapsing-remitting MS, clinically isolated syndrome and active secondary progressive MS. Although it competes with Gilenya (fingolimod) and Mayzent (siponimod), the sphingosine-1-phosphate receptor modulator does not require first-dose monitoring for bradycardia (a slow heart rate).
Evrysdi (risdiplam). Approved in August, Evrysdi is the first oral drug approved to treat spinal muscular atrophy (SMA). The two other drugs approved for some patients with SMA are Spinraza (nusinersen), administered as an intrathecal injection once every four months, and Zolgensma (onasemnogene abeparvovec-xioi), a gene therapy administered as a one-time IV injection.
Some notable 2020 FDA approvals of new oncology drugs
Trodelvy (sacituzumab govitecan-hziy). Approved in April, Trodelvy is an antibody-drug conjugate for the treatment of patients with metastatic triple-negative breast cancer who received at least two prior therapies. The antibody “finds” the cancer, and the drug attacks the cancer.
Zepzelca (lurbinectedin). Approved in June, Zepzelca is an alkylating drug for adult patients with metastatic small cell lung cancer who have progressed on or after chemotherapy.
Sarclisa(isatuximab-irfc). Approved in March, Sarclisa is a monoclonal antibody that binds to the CD38 receptor on multiple myeloma cells. The FDA approval is for Sarclisa in combination with pomalidomide and dexamethasone for adult patients with multiple myeloma who have received at least two prior therapies.
Tabrecta (capmatinib). Approved in May, Tabrecta is a kinase inhibitor used to treat metastatic non-small cell lung cancer. It is one of an increasing number of agents that home in on cancer cells with a particular mutation, in this case a mutation that leads to MET exon 14 skipping.
Every nook and cranny of American healthcare has been affected by the COVID-19 pandemic, and drug development and approvals are no exception. But, remarkably, the number of drug approvals coming from the FDA showed no signs of slowing down this year, with 47 novel drugs approved as of the beginning of December, among them a record number of oncology drugs. Judging by Prescription Drug User Fee Act dates — dates by which the FDA is supposed to complete its review of an application — it looks like the number of FDA-approved new drugs this year will easily surpass the 48 approved last year but will probably fall short of the record of 59 approvals two years ago.
The COVID-19 effect
Does that mean that drug development escaped unscathed? No. As FDA drug approvals rolled in during the first half of the year, drug developers and those dependent on the pharmaceutical industry fretted that the number of approvals would fall off sharply as the year went on and the pandemic continued. In March, when the pandemic hit the United States, several drug companies moved to halt or delay new studies and patient enrollment.
But there was still some pre-pandemic momentum to the drug pipeline. If the pandemic has slowed down drug development and approvals, it will likely become more evident next year and the year after. Investigators are still trying to figure out safe, reliable ways to enroll and monitor patients in clinical trials, although steps have already been taken to do some trials remotely.
Looking at it from another perspective, the pandemic hasn’t crimped the drug pipeline so much as tested how fast it can expand and adjust as companies — with government assistance — raced to develop treatments and vaccines for a disease that didn’t exist before this year.
It hasn’t been all smooth sailing. According to many experts, the FDA emergency use authorizations (EUAs) for hydroxychloroquine, the drug touted by President Donald Trump, and convalescent plasma were a mistake — and in June the FDA did wind up revoking the EUA for chloroquine and hydroxychloroquine. The wisdom of full-fledged approval for Gilead’s Veklury (remdesivir) has also been questioned. On the other hand, as we went to press, the FDA was considering emergency use authorizations for two COVID-19 vaccines, one developed by Pfizer and BioNTech, a German company, and the other by Moderna, a biotech company in Cambridge, Massachusetts.
Specialty dominates
Meanwhile, in the “normal,” non-COVID-19 pipeline, there has been a marked shift recently in the types of drugs the FDA approves. The agency is approving more specialty drugs — expensive drugs for relatively rare diseases — than so-called traditional drugs. In fact, over the past five years, two-thirds of FDA approvals have been for specialty drugs, and 2020 is no exception, says Aimee Tharaldson, Pharm.D., senior clinical pharmacist of Emerging Therapeutics at Express Scripts. So far, 68% of 2020 approvals have been for specialty drugs.A large proportion of new drug approvals this year have been for oncology drugs. So far, 18 novel oncology drugs have gotten the nod from the FDA, and a few more may be approved before the year is over. According to Tharaldson, the oncology approvals this year break the record number of 17 approved in 2018.
Good time for orphans
The FDA is also approving orphan drugs at steady clip; Tharaldson notes that more than half of the new drugs approved so far in 2020 are in that category. Orphan drugs are a subset of specialty drugs that the FDA puts on a separate approval track that makes the developers eligible for a variety of incentives.
Two of the notable orphan drug approvals are for neuromyelitis optica spectrum disorder, a rare autoimmune disease that affects approximately 10,000 people in the United States. The disease, which can be misdiagnosed as multiple sclerosis, is characterized by inflammation of the optic nerve and spinal cord. Severe cases, can lead to blindness and impaired mobility. In June, the FDA approved Uplizna (inebilizumab-cdon), a CD19-directed antibody and, in August, Enspryng (satralizumab-mwge), an interleukin 6 inhibitor.
It is a different story with generics. According to Regulatory Focus, the online publication of the Regulatory Affairs Professionals Society, 2020 did not bode well for future generic competition. In fiscal year 2020, the FDA approved 909 abbreviated new drug applications (ANDAs) for generics, a 22% drop from fiscal year 2019, when the FDA approved 1,171 ANDAs.
Jaime Rosenberg is a freelance writer based in Jersey City,New Jersey.
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