DNDi - João Roberto RipperThe GARDP neonatal sepsis programme aims to provide an evidence base for the use of antibiotics, both old and new, in neonates with serious bacterial infections (SBIs), as the currently available standard of care in many countries is increasingly becoming less effective due to antimicrobial resistance. Other long-term objectives include the development of formulations that are adapted for use in neonates, as well as collaborations with WHO and other relevant stakeholders to ensure that the R&D innovation of new treatments encompasses a solid strategy for stewardship, and affordable and sustainable access.

 

GARDP’s neonatal sepsis programme aims to:

  • Deliver new treatment regimens for babies with neonatal sepsis;
  • Establish a global expert advisory group to tackle key issues in neonatal sepsis case management and treatment;
  • Set up a global network of specialist centres focusing on the conduct of studies to provide an evidence base for the use of antibiotics in neonatal infectious diseases;
  • Design and conduct pharmacokinetic, observational and interventional studies to determine the efficacy and safety of new treatment regimens in neonates for the optimal use of old, off-patent and new antibiotics; and
  • Develop a solid stewardship and access strategy for new treatment regimens.

This programme aligns with the SDG of reducing under-five mortality.


 

By 2023, GARDP aims to deliver one first-line combination treatment for clinically diagnosed cases of neonatal sepsis and initiate late phase trials evaluating one combination treatment for cases of confirmed multidrug-resistant gram-negative bacteria.

 

More information:

Read the programme strategy

Read a snapshot of the programme: English  Deutsch

 

 

Indication

Empiric treatment of neonatal sepsis, including meningitis (premature and term, early and late onset)

Patient population

Neonates with pSBI in settings of high prevalence of resistance to first line WHO empiric therapy

Route of administration

i.v. (intravenous), 30-120 min infusions

Dosing schedule

2-4 x daily

Efficacy

Comparable clinical activity to amoxicillin/gentamicin or ceftriaxone/gentamicin in claimed indication

Clinical activity in pathogens resistant to amoxicillin/gentamicin or ceftriaxone/gentamicin

Treatment duration

5-28 days

Safety/ Tolerability

Low propensity for resistance development, large therapeutic window concerning hepatotoxicity, nephro- and CNS-toxicity, no QT-prolongation

Drug interactions

Comparable to competitors

Key countries

Europe, the Americas, Asia, Africa

Price/ Day of therapy

Average ex-factory price at launch: low/DOT (directly observed therapy)

Pharmacoeconomics

Reduction of intensive care unit and hospitalization days (modelling). Reimbursable

Main competitors

Amoxicillin/gentamicin or ceftriaxone/gentamicin

Indication

Neonatal sepsis, where MDR Gram-negative pathogens have been demonstrated, including K. pneumoniae, P. aeruginosa or Acinetobacter spp. Including CROs

Neonatal meningitis

Patient population

Hospitalized neonates with severe infections, failure on optimal current  treatment and proven microbiology

Route of administration

i.v. (intravenous), 30-120 min infusions

Dosing schedule

2-4 x daily

Efficacy

Comparable clinical activity to existing options in claimed indication

Clinical activity in pathogens resistant to carbapenems

Treatment duration

5-28 days

Safety/ Tolerability

Low propensity for resistance development, large therapeutic window concerning hepatotoxicity, nephro- and CNS-toxicity, no QT-prolongation

Drug interactions

Comparable to competitors

Key countries

Europe, the Americas, Asia, Africa

Price/ Day of therapy

Average ex-factory price at launch: tbd/DOT (directly observed therapy)

Pharmacoeconomics

Reduction of intensive care unit and hospitalization days (modelling). Reimbursable

Main competitors

Colistin monotherapy


 

February 2018 Update

Progress to date includes developing two target product profiles (TPPs) and building solid partnerships and multi-stakeholder collaborations. These include St George’s, University of London to conduct an observational study and input into the pharmacokinetic (PK) and pharmacodynamic (PD) assessment of potential drug candidates and the PENTA Foundation.

Discussions are ongoing to identify a partner to coordinate and monitor microbiological activities including determining the phenotypic and genotypic resistance profiles of invasive and colonizing Gram-negative isolates.

A scoping study of existing drugs and the drug pipeline is ongoing to identify treatments that meet the predefined criteria of the TPPs. The initial feasibility survey, conducted in 2017, confirms the critical need and high levels of drug resistance with significant variation in treatment protocols in place in countries. Protocols for an observational and clinical PK study are ready for implementation in early 2018. A stewardship and access plan is under development.