The ID Pipeline: FDA Activity From the Week of June 30, 2019

Here is a look at infectious disease-related US Food and Drug Administration (FDA) news from the week of June 30, 2019.

FDA Accepts NDA for RedHill Biopharma's RHB-105 for the Treatment of H pylori

On Wednesday, July 3, 2019, the FDA accepted for review RedHill Biopharma Ltd.'s New Drug Application for RHB-105 (Talicia®) for the treatment of H pylori infection and granted Priority Review designation. A target Prescription Drug User Act (PDUFA) action date of November 2, 2019, has been assigned.

RHB-105 is a novel and proprietary fixed-dose, all-in-one oral capsule combination of 2 antibiotics—rifabutin and amoxicillin—and a proton pump inhibitor, omeprazole.

“The efficacy of current standard-of-care therapies for H pylori bacterial infection continues to decline due to the high and steadily increasing prevalence of antibiotic-resistant strains. This public health concern is widely recognized by the US government and other leading health agencies, including the World Health Organization, which classified H pylori as a Group I carcinogen and highlighted the urgent need for new treatments for H pylori eradication,” Dror Ben-Asher, Chief Executive Officer of RedHill Biopharma, said in a press release. “The acceptance for review of the NDA for Talicia and the Priority Review designation are encouraging steps as we continue our intensive preparations for a potential US commercial launch of Talicia as a new first-line, standard-of-care therapy for H pylori. We continue to work closely with the FDA, with the aim of bringing this important new therapy to patients.”

The full press release is available here.

Genentech Announces Positive Phase 3 Study Results Evaluating 1-Dose Baloxavir Marboxil

On Tuesday, July 2, 2019, Genentech announced positive results for its phase 3 MINISTONE-2 study on 1-dose baloxavir marboxil for children with influenza.

According to the study, which met its primary end point, the treatment, given as a new oral suspension, is a well-tolerated and effective potential treatment for the flu in children aged 1 to less than 12 years.

“Children need new medicines for the flu because they are at higher risk of developing the flu and more likely to have complications such as breathing problems and pneumonia. These flu complications, which in some cases can be fatal, lead to approximately 1 million children under 5 being admitted to the hospital globally every year,” Sandra Horning, MD, chief medical officer and head of Global Product Development, said in a press release. “As a 1-dose oral suspension medicine, Xofluza could potentially provide a convenient treatment option for children with the flu, and we look forward to sharing these data with health authorities around the world.”

The full press release is available here.

Valneva Launches Phase 2 Study for Lyme Disease Vaccine Candidate

On Monday, July 1, 2019, Valneva SE announced initiation of a second phase 2 study for VLA15 to determine optimal dosage levels and vaccination schedule for phase 3 trials of the Lyme vaccine candidate.

VLA15-202, a randomized, observer-blind, placebo controlled trial, will deliver 1 of 2 dosage levels to 250 subjects.

“We are pleased to continue to progress our Lyme vaccine candidate development according to plan and as expeditiously as possible," Wolfgang Bender, MD, PhD, Chief Medical Officer of Valneva, said in a press release. "The disease footprint is widening and the need for a vaccine to prevent this significant unmet medical need is increasing. With higher dosage levels and the potential alternative vaccination schedule, our ultimate goal is to further optimize our vaccine candidate by targeting a high efficacy from the first Lyme season.”

The FDA granted the program Fast Track designation in July 2017.

The full press release is available here.