FDA watch: New drug approvals in 2019

By John Murphy, MDLinx
Published May 10, 2019

Key Takeaways

The FDA approved 59 new drugs in 2018, well above the yearly average of 33 new drug approvals. And the agency continues to be busy in 2019, with 10 novel drugs approved so far. But it’s not easy to keep up with all these new drugs. To help with that, here’s a list of some of the most notable new approvals this year.

Zulresso for postpartum depression

Zulresso (brexanolone) is the first drug specifically approved for the treatment of postpartum depression in adult women. The FDA designated it a Breakthrough Therapy and granted it Priority Review.

Zulresso is administered as a continuous intravenous (IV) infusion over the course of 60 hours (2.5 days). Because the drug can cause excessive sedation or sudden loss of consciousness during infusion, it can only be administered in certified healthcare facilities where the patient can be carefully monitored.

In two placebo-controlled clinical trials involving women with moderate to severe postpartum depression, Zulresso showed better reduction of depressive symptoms than placebo. Reduction in depressive symptoms began as early as 24 hours and continued throughout the 30-day follow-up period. 

Balversa for metastatic bladder cancer

The FDA granted accelerated approval to Balversa (erdafitinib), the first targeted therapy for metastatic bladder cancer. The drug is indicated for the treatment of adults with locally advanced or metastatic urothelial carcinoma with susceptible fibroblast growth factor (FGFR) genetic alterations.

FGFRs regulate biological processes, including cell growth and division, during development and tissue repair. Genetic alterations in FGFRs occur in roughly one in five patients with recurrent and refractory bladder cancer. Balversa is the first FGFR kinase inhibitor approved by the FDA. It’s indicated in patients whose bladder cancer has progressed despite prior platinum-containing chemotherapy.

In one clinical trial that included patients with locally advanced or metastatic bladder cancer with FGFR3 or FGFR2 genetic alterations, the overall response rate to Balversa treatment was 32.2%. Of these patients, 2.3% had complete response and almost 30% had partial response. The response lasted approximately 5 ½ months on average. Some of the patients who responded to Balversa had not responded to prior anti-programmed death-ligand 1 (PD-L1)/PD-1 therapy.

Serious adverse effects included ocular disorders, increased phosphate level, and embryo-fetal toxicity. Ocular disorders included central serous retinopathy/retinal pigment epithelial detachment, which was reported in 25% of treated patients. Dry eye symptoms occurred in 28% of treated patients.

To detect FGFR genetic alterations in the tumor tissue of patients with metastatic bladder cancer, the FDA also approved the therascreen FGFR RGQ Reverse-Transcription Polymerase Chain Reaction Kit for use as a companion diagnostic test with Balversa.

Benlysta for lupus in children

Following Priority Review, the FDA approved Benlysta (belimumab) IV infusion, the first treatment for lupus (systemic lupus erythematosus) in children ages 5 and older. Benlysta was approved in 2011 for adults with lupus.

Although lupus is uncommon in children, it puts them at a higher risk than adults for developing increased organ damage and complications, not to mention the adverse events from life-long treatment.

In a 1-year clinical trial, pediatric patients with lupus who were treated with Benlysta IV plus standard therapy had a clinically higher improvement in disease activity (52.8%) compared with pediatric patients who received standard therapy alone (43.6%). In addition, those who received Benlysta IV plus standard therapy also had a lower risk for severe flares, as well as longer periods between severe flares (160 days vs 82 days).

The drug’s safety and pharmacokinetic profiles in children were consistent with those in adults with lupus. Also, based on data from clinical studies in adults with lupus, Benlysta’s doctor and patient information includes a warning for mortality, serious infections, hypersensitivity, and depression.

Spravato for treatment-resistant depression

Spravato (esketamine) nasal spray is indicated for adults with treatment-resistant depression. It’s meant to be used in conjunction with an oral antidepressant. This marks the first approval for any use of esketamine, and the FDA gave it Fast Track status as a Breakthrough Therapy.

In one short-term clinical trial, Spravato reduced the severity of depression within 2 days. However, in two other short-term trials, the drug didn’t meet the pre-specified level of effectiveness. In a longer-term maintenance-of-effect trial, patients who continued to use Spravato with an oral antidepressant had a longer time before relapse of depressive symptoms than patients on an oral antidepressant alone.

Because the drug can make patients feel sedated or detached, patients can’t use the spray at home. It must be self-administered in a certified doctor’s office or clinic under the supervision of a physician or other healthcare provider.

Evenity for osteoporosis

Evenity (romosozumab-aqqg) is indicated for treating osteoporosis in postmenopausal women at high risk of bone fracture. It’s the first osteoporosis drug that both increases bone formation and, to a lesser extent, reduces bone loss.

The drug is injected in two consecutive shots once a month by healthcare professional. However, after 12 months of injections, the drug’s bone-forming effect wanes, so treatment should be discontinued. Patients who need further therapy should begin an osteoporosis treatment that reduces bone breakdown.

In a clinical trial, 1 year of Evenity treatment lowered the risk of new vertebral fracture by 73% compared with placebo. In another trial, 1 year of Evenity followed by 1 year of another osteoporosis therapy (alendronate) reduced the risk of a new vertebral fracture by 50% compared with 2 years of alendronate treatment alone. Evenity followed by alendronate also reduced the risk of nonvertebral fractures compared with alendronate alone.

The drug’s label carries a boxed warning because Evenity may increase the risk of heart attack, stroke, and cardiovascular death. So, it shouldn’t be used in patients who’ve had heart attacks or strokes within the previous year. “Health care professionals should also consider whether the benefits of Evenity outweigh its risks in those with other risk factors for heart disease and should discontinue Evenity in any patient who experiences a heart attack or stroke during treatment,” according to the FDA.

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