FDA approvals to watch for in 2020

By John Murphy, MDLinx
Published December 4, 2019

Key Takeaways

A number of novel drugs are expected to gain FDA approval in 2020—and one of them has already been approved, weeks before the New Year.

In late November—at least 3 months earlier than expected—the FDA approved a drug to treat sickle cell disease. The drug is voxelotor (Oxbryta, Global Blood Therapeutics), a once-daily oral medication that is the first treatment to directly target the root cause of sickle cell disease: sickle hemoglobin polymerization, or the “sickling” of red blood cells. The FDA granted the drug priority review, fast track, and breakthrough therapy designations. But voxelotor won’t be cheap—it’s expected to cost more than $10,000 per month.

But that’s not the only promising drug to look forward to. Here’s what to watch for in medicines that are expected to receive FDA approval in 2020.

Aducanumab for Alzheimer disease

In March 2019, biotech company Biogen halted phase 3 trials of its novel Alzheimer disease drug aducanumab because results indicated that the drug had no significant effects on clinical symptoms such as memory loss and disorientation. But in late October, the company announced that it would seek FDA approval for aducanumab based on a re-evaluation of the phase 3 data.

In this new analysis, which included additional patient data, the drug met its primary endpoint: a significant reduction in cognitive decline. Patients who received aducanumab experienced significant benefits on measures of cognition and function such as memory, orientation, and language, and also had improvements in activities of daily living.

Biogen stated that if aducanumab is approved, it would become the first therapy to reduce the clinical decline of Alzheimer disease, and would also be the first therapy to demonstrate that targeting amyloid beta proteins in the brain results in better patient outcomes.

Ubrogepant for acute migraine

Although several medications (such as triptans) are available for the acute treatment of migraine, they’re contraindicated in many patients, and cause adverse effects in others. In some patients, they’re simply ineffective.

Now, investigators showed in a recent phase 3 trial that ubrogepant, given as a 25- or 50-mg oral pill, significantly reduced migraine pain within 2 hours compared with placebo. In addition, the 50-mg pill reduced the most bothersome migraine symptoms, such as photophobia, phonophobia, and nausea. Both doses were well tolerated, with adverse events similar to placebo.

Ubrogepant, made by Allergan, is an oral calcitonin gene-related peptide receptor antagonist—a new family of drugs known as gepants.

Cabotegravir/rilpivirine for HIV

The current standard of care for treating HIV is a daily, oral, three-drug regimen of antiretroviral therapy. But, investigators have now shown in two phase 3 trials that a once-monthly injection of cabotegravir/rilpivirine (Cabenuva, ViiV Healthcare) is as effective as the standard daily treatment. If approved, this would be the first long-acting, injectable treatment for adults living with HIV, potentially changing the way many patients living with HIV are managed and treated. In the clinical trials, up to 90% of patients preferred the long-acting injectable regimen compared with those who preferred their previous oral therapy.

The FDA has granted priority review to cabotegravir/rilpivirine, with a target decision date of December 29, 2019.

Ozanimod for multiple sclerosis

Ozanimod could be a first-line oral treatment option for people with relapsing multiple sclerosis (MS), and one with fewer side effects than conventional treatment.

In recent phase 3 clinical trials, results of which are published in The Lancet Neurology, ozanimod was significantly more effective than interferon beta-1a injection, the current first-line treatment for relapsing MS. Investigators showed that people with relapsing MS treated with once-daily oral ozanimod had reduced disability progression, fewer MS relapses, and a decrease in the number of active brain lesions compared with those treated with weekly interferon beta-1a injections. Importantly, ozanimod was well tolerated, with fewer treatment-related adverse events.

Ozanimod, produced by Celgene, is currently under FDA review, with a decision expected by March 25, 2020.

Teprotumumab for thyroid eye disease

The FDA gave teprotumumab priority review, orphan drug, fast track, and breakthrough therapy designations. If approved, it would be the first medicine for the treatment of active thyroid eye disease.

Produced by Horizon Therapeutics, teprotumumab is a fully human monoclonal antibody inhibitor of insulin-like growth factor 1 receptor (IGF-1R). In a phase 3 clinical trial, investigators showed that the drug provided significant reductions in the effects of active thyroid eye disease—including inflammation, diplopia, and clinical activity score—and improved quality of life compared with placebo. Notably, 77.4% of patients on teprotumumab had an average 2.63-mm reduction in proptosis (bulging of the eyes).

Teprotumumab is currently under review by the FDA, with an expected decision date of March 8, 2020.

Trastuzumab deruxtecan for HER2-positive metastatic breast cancer

Trastuzumab deruxtecan is a promising new medicine for the treatment of human epidermal growth factor receptor 2 (HER2)-positive metastatic breast cancer. Developed through a collaboration between AstraZeneca and Daiichi Sankyo, trastuzumab deruxtecan is an HER2-targeting antibody drug conjugate.

The companies’ application to the FDA was based on results from a phase 1 study published in The Lancet Oncology, as well as results from a phase 2 study that will be presented in detail at the upcoming San Antonio Breast Cancer Symposium this month. In the phase 1 study, although all patients had at least one treatment-emergent adverse event, nearly 60% achieved a confirmed objective response.

The FDA has granted the drug priority review, with an expected decision date in the second quarter of 2020.

Dextromethorphan/bupropion for depression

The FDA granted breakthrough therapy designation to AXS-05 (dextromethorphan/bupropion modulated delivery tablet, Axsome) for the treatment of major depressive disorder (MDD), as well as fast track designation for treatment-resistant depression (TRD).

In AXS-05, dextromethorphan acts as a glutamate receptor modulator—a novel mechanism of action—as well as a sigma-1 receptor agonist, a nicotinic acetylcholine receptor antagonist, and an inhibitor of serotonin and norepinephrine transporters. The bupropion component increases the bioavailability of dextromethorphan, serves as a norepinephrine and dopamine reuptake inhibitor, and is a nicotinic acetylcholine receptor antagonist.

Axsome is expected to report topline results of AXS-05 phase 3 trials for MDD before the end of 2019, and topline results of an AXS-05 trial for TRD in the first quarter of 2020. If these results are successful, the company expects to submit a new drug application for AXS-05 in the second half of 2020.

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