Biggest FDA approvals of 2019

The FDA approved an avalanche of 59 drugs in 2018. The agency slowed things down for 2019, with 42 approved drugs as of this writing. But what this list of new drugs may lack in quantity, it makes up for in novelty, innovation—and cost. One newly approved treatment is reported to be the most expensive drug ever sold. 

Here’s the latest on that pricey drug, as well as several other new drug approvals that made a splash this year. 

Zolgensma for spinal muscular atrophy

In late May, the FDA approved Zolgensma (onasemnogene abeparvovec-xioi, AveXis), the first gene therapy for treating infants and children under 2 years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality. 

News of the gene therapy’s approval was quickly overshadowed by controversy over its cost. The one-time infusion of Zolgensma has a sticker price of $2.1 million. 

The drug made headlines again in August when AveXis revealed that its investigators had manipulated preclinical data from tests performed in animals. Worse yet, the company knew about the data manipulation more than 2 months before the FDA approved the drug, but didn’t reveal this to the agency until a month after Zolgensma’s approval. 

The FDA announced an investigation, but also stated that the drug should remain on the market because human clinical trial data supported its efficacy. 

Trikafta for cystic fibrosis 

In late October, the FDA approved Trikafta (elexacaftor/ivacaftor/tezacaftor, Vertex Pharmaceuticals), the first triple combination therapy to treat patients age 12 years and older who have the most common cystic fibrosis genetic mutation, representing an estimated 90% of people with the disease. 

In a phase 3 clinical trial, patients on Trikafta had an average 14% increase in lung capacity (measured by forced expiratory volume in 1 second) compared with those on placebo. 

“This should be a cause for major celebration,” wrote Francis S. Collins, MD, PhD, director of the NIH, in an editorial. Dr. Collins co-led the research that, in 1989,  identified the gene that causes cystic fibrosis.

Trikafta will cost about $312,000 annually.

Zulresso for postpartum depression

Zulresso (brexanolone, Sage Therapeutics) is the first FDA-approved drug specifically for the treatment of postpartum depression, which affects about one in nine US mothers.

In two clinical trials, postpartum women treated with Zulresso had significantly reduced depression scores compared with those taking placebo, and the effects occurred quickly—within 2 or 3 days—and lasted at least 1 month. 

The drug is administered as a continuous IV infusion over 2.5 days, which must be performed by a healthcare provider in a certified facility. Patients must be monitored for excessive sedation and sudden loss of consciousness along with continuous pulse oximetry. The list price for Zulresso is about $34,000 per course of therapy.

Oxbryta for sickle cell disease

In late November, the FDA approved Oxbryta (voxelotor, Global Blood Therapeutics) for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. The drug is a once-daily oral medication that’s the first treatment to directly target the root cause of sickle cell disease: sickle hemoglobin polymerization, or the “sickling” of red blood cells. 

In a clinical trial that included 274 patients with SCD, those who received 1,500 mg of Oxbryta had a 51.1% hemoglobin response rate compared with 6.5% for those who received placebo. But that kind of efficacy appears to come at a hefty price—the drug is expected to cost more than $10,000 per month, and must be taken for life.

Mayzent for multiple sclerosis

Mayzent (siponimod, Novartis), approved in late March, is an oral drug indicated for adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive MS (SPMS). 

It’s the first drug to treat patients with active SPMS, which is meaningful because up to 80% of patients with relapsing-remitting MS develop SPMS. 

In a phase 3 clinical trial—the largest of its kind in patients with SPMS—siponimod reduced the risk of disability progression by 21% over 3 months and demonstrated a safety profile on par with immunomodulators currently used for relapsing MS. Its list price is $88,500 per year.

Skyrizi for psoriasis 

AbbVie already has one blockbuster drug for treating plaque psoriasis: Humira (adalimumab). But in late April, the FDA approved the company’s Skyrizi (risankizumab-rzaa), an interleukin-23 inhibitor for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy. 

In a head-to-head phase 3 clinical trial of risankizumab and adalimumab, researchers found that 72% of patients on risankizumab achieved clear or nearly clear skin at 16 weeks of follow-up compared with 47% of patients given adalimumab. AbbVie launched the drug in May with a list price of $59,000 for a year’s treatment.