The ID Pipeline: FDA Activity From the Week of September 22, 2019

Here is a look at infectious disease-related US Food and Drug Administration (FDA) news from the week of September 22, 2019.

Phase 3 LEAP 2 Results Published Following FDA Approval of Lefamulin for Community-Acquired Bacterial Pneumonia

On Friday, September 27, 2019, Nabriva Therapeutics announced the publication of Lefamulin Evaluation Against Pneumonia 2 (LEAP 2) study results following FDA approval of lefamulin (Xenleta) for the treatment of community-acquired bacterial pneumonia (CABP) last month.

The phase 3 clinical trial results were published in JAMA.

“The increasing prevalence of antimicrobial resistance in the most common causative pathogens of CABP, along with growing evidence of safety concerns with fluoroquinolones makes a compelling case for the development of new antibacterial classes with novel mechanisms of action (MOA) to treat patients with CABP,” Jennifer Schranz, MD, Chief Medical Officer of Nabriva Therapeutics, said in a press release. “Xenleta is the first novel MOA antibiotic approved in the United States in nearly 20 years. The results from the LEAP clinical trial program, combined with lefamulin’s targeted spectrum of activity, which aligns with the principles of antimicrobial stewardship, make it an attractive short-course, monotherapy treatment option for adults with CABP.”

The full press release is available here.

FDA Expands Approval for Glecaprevir and Pibrentasvir

On Thursday, September 26, 2019, the FDA approved expanded the approval of glecaprevir and pibrentasvir (Mavyret). Under the expansion, the tablets are now approved for an 8-week duration for treatment-naïve children ages 12 years and older and adults who have chronic hepatitis C virus (HCV) genotype 1, 2, 3, 4, 5 or 6 infection and compensated cirrhosis. The approval was granted to AbbVie Inc.

According to the US Centers for Disease Control and Prevention, between 2.7 and 3.9 million people in the United States have chronic HCV, and children born to HCV-positive mothers are at risk for HCV infection. It is estimated that there are 23,000 to 46,000 children in the United States with HCV infection.

The FDA's statement indicates that glecaprevir and pibrentasvir is now the first 8-week treatment approved for adults and certain pediatric patients with HCV genotypes 1-6 without cirrhosis and with compensated cirrhosis. Previously, standard treatment for patients with compensated cirrhosis was at least 12 weeks.

“This approval provides a treatment duration of 8 weeks for both pediatric and adult patients with compensated cirrhosis regardless of HCV genotype; meaning that an 8-week treatment regimen is available for any treatment-naïve HCV patient, regardless of cirrhosis status or genotype,” said Jeffrey Murray, MD, deputy director of the Division of Antiviral Products in the FDA’s Center for Drug Evaluation and Research, in the announcement. “Mavyret is a combination of direct-acting antiviral drugs that reduce the amount of HCV in the body to undetectable levels by preventing the virus from multiplying, and in most cases, curing HCV infection.”

The full story is available here.

Vaxart Inc Announces Positive Topline Results From Phase 1b Study of Oral Bivalent Norovirus Vaccine

On Wednesday, September 25, 2019, Vaxart, Inc. announced topline results from its Phase 1b safety, immunogenicity, and interference study of its oral tableted bivalent norovirus vaccine for healthy adults.

The randomized, double-blind, placebo-controlled trial met all primary endpoints for safety and demonstrated robust immunogenicity, with 78%-93% of subjects responding by eliciting IgA antibody secreting cells, a key marker for mucosal immunity and a potential correlate of protection for norovirus disease.

“Norovirus is a very contagious disease which can be especially harmful to children under age 5 as well as to older adults,” William Schaffner, MD, medical director of the National Foundation for Infectious Diseases (NFID) and professor of preventive medicine and infectious diseases at Vanderbilt University School of Medicine, said in a press release. “An effective and safe vaccine would be critical to protecting high-risk populations against serious illness caused by norovirus, especially if it could be administered orally.”

The full press release is available here.

FDA Approves Smallpox, Monkeypox Vaccine

On Tuesday, September 24, 2019, the FDA approved the first live, non-replicating vaccine indicated for the prevention of smallpox and monkeypox.

The Jynneos Smallpox and Monkeypox Vaccine, Live, Non-Replicating, from Bavarian Nordic A/S, was approved for preventing smallpox and monkeypox in adults aged 18 years or older who have been determined to be at high risk for either infection.

With the indication, it also becomes the only FDA-approved vaccine for the prevention of monkeypox—a rare virus infection with similar, mild symptoms to smallpox. The possibly fatal disease last made headlines in the US during a 2003 outbreak, when the first human case was reported outside of Africa.

The vaccine will be made available for clinically determined high-risk patients, and will also be included in the Strategic National Stockpile, the US’ greatest surplus of pharmaceuticals and medical supplies for us in potentially severe public health emergencies.

The full story is available here.