The ID Pipeline: FDA Activity From the Week of August 4, 2019

Here is a look at infectious disease-related US Food and Drug Administration (FDA) news from the week of August 4, 2019.

T2Bacteria Panel Granted New Technology Add-on Payment by CMS

On Tuesday, August 6, 2019, T2 Biosystems Inc. was granted a New Technology Add-on Payment (NTAP) from the US Centers for Medicare & Medicaid Services (CMS) for the T2Bacteria® Panel for 2020, making it the first-ever in-vitro diagnostic test to receive the approval, which is reserved for new technologies that demonstrate substantial clinical improvement.

“By approving NTAP for the T2Bacteria Panel, CMS has confirmed that the T2Bacteria Panel represents a substantial clinical improvement over existing technologies,” John McDonough, chairman and chief executive officer at T2 Biosystems, said in a press release. “The introduction of the T2Bacteria Panel has altered the course of clinical treatment in a number of ways, and this new designation will ensure increased access to this groundbreaking, direct-from-blood rapid diagnostic for patients suspected of a bloodstream infection or sepsis.”

"The T2Bacteria Test Panel represents a substantial clinical improvement over existing technologies because it reduces the proportion of patients on inappropriate therapy, thus reducing the rate of subsequent diagnostic or therapeutic intervention as well as length of stay and mortality rates caused by sepsis causing bacterial infections," CMS said in a statement.

The full press release is available here.

Positive Topline Results Announced in Basilea's Phase 3 TARGET Study of Ceftobiprole for ABSSSI

On Tuesday, August 6, 2019, Basilea Pharmaceutica Ltd. announced positive topline results in its Phase 3 TARGET study evaluating ceftobiprole in the treatment of patients with acute bacterial skin and skin structure infections.

A total of 679 patients were enrolled in the randomized, double-blind, multicenter TARGET study, where ceftobiprole met the primary efficacy objective of non-inferiority compared with vancomycin plus aztreonam in the intent-to-treat population. Ceftobiprole was well-tolerated and the rate of adverse events were similar between the 2 treatment groups (20% ceftobiprole vs. 18% vancomycin plus aztreonam).

Basilea is also exploring a second indication for ceftobiprole to treat Staphylococcus aureus bloodstream infections. If the results of both studies are positive, Basilea plans to submit a New Drug Application to the FDA.

"The successful completion and positive results of our phase 3 study in skin infections marks the achievement of a significant milestone towards a filing of ceftobiprole in the US," Marc Engelhardt, PhD, chief medical officer of Basilea, said in a press release. "The second study, in S aureus bloodstream infections, is well on track and is expected to deliver topline results in the second half of 2021. In both indications, ceftobiprole addresses unmet medical needs through its broad-spectrum rapid bactericidal activity, with the ability to cover both gram-positive and gram-negative pathogens and with the well-established safety profile of a cephalosporin."

The full press release is available here.

FDA, Novavax Reach Agreement on Phase 3 Trial Design for NanoFlu Treatment for Adults Aged 65 and Over

On Monday, August 5, 2019, the FDA and Novavax, Inc. reached an agreement on a Phase 3 trial design for Nanoflu™, an adjuvanted recombinant quadrivalent seasonal influenza vaccine candidate for older adults aged 65 and over.

The Phase 3 clinical trial is expected to launch in the fall of 2019, with topline clinical data expected in the first quarter of 2020. Novavax plans to use the results to bolster a Biologics License Application and licensure of NanoFlu using the accelerated approval pathway.

“With CDC-reported overall influenza vaccine effectiveness of just 12 percent among older adults during the 2018-2019 season, the pressing need for a more effective vaccine in this population is clear,” Stanley C. Erck, president and chief executive officer of Novavax, said in a press release. “We are committed to quickly demonstrating that our recombinant nanoparticle approach can help close the gap that exists between circulating flu strains and vaccines produced by traditional methods that frequently result in a mismatch, and ultimately, leave millions vulnerable to the serious consequences of influenza disease.”

The full press release is available here.

CytoDyn's BLA Filing for Leronlimab (PRO 140) Granted Small Business Waiver of Application Fees

On Monday, August 5, 2019, the FDA granted CytoDyn Inc. a small business waiver of application fees for the pending Biologics License Application (BLA) filing for leronlimab (PRO 140) as a combination therapy with HAART for people living with HIV.

The FDA previously granted CytoDyn "Fast Track" designation for 2 potential indications for leronlimab: as combination therapy with HAART for people living with HIV, and for metastatic triple-negative breast cancer (mTNBC).

“We are extremely grateful to the FDA for granting the fee waiver,” Nader Pourhassan, PhD, CytoDyn president and CEO, said in a press release. “Our goal firmly remains to complete in September 2019 the BLA filing with the FDA for leronlimab as a combination therapy with HAART for HIV-infected patients and hopefully initiate our first monotherapy pivotal trial for label expansion soon after our in-person meeting with the FDA. We are also hopeful to initiate our funded PrEP clinical trial this year, which will be conducted in Thailand.”

The full press release is available here.