# Accelerated Development of Lofexidine for Neonatal Opioid Withdrawal Syndrome

> **NIH NIH R01** · US WORLDMEDS, LLC · 2020 · $800,000

## Abstract

PROJECT SUMMARY:
Over the past decade, rates of opioid use among the general population has continued to rise in the United
States. This astounding increase in opioid use has resulted in the government declaring a public health
emergency in 2017. Compounding the problem, a concurrent rise in opioid use among pregnant females has
resulted in a massive increase in infants born suffering from opioid withdrawal, termed Neonatal Opioid
Withdrawal Syndrome (NOWS). The NOWS epidemic has led to significant increases in healthcare costs and
burden on the healthcare system due to increased length of hospital stay and level of required care. A normal
infant averages 2.1 days of hospital care post-birth costing $3,500, whereas an infant born with NOWS averages
16.9 days of inpatient care costing $66,700. Currently, there are no FDA-approved medications available for
treatment of NOWS. US WorldMeds (USWM) has developed lofexidine in collaboration with NIDA for the
treatment of opioid withdrawal in adults, and anticipates possible Food and Drug Administration (FDA) approval
for this use in May 2018. USWM now proposes to extend this successful co-development program through
investigation of the safety and efficacy of lofexidine in NOWS. This application proposes a strategic alliance
between USWM and the National Institute on Drug Abuse (NIDA) to complete development and registration
prerequisites prior to initiation of clinical assessment of lofexidine in the neonatal patient population.
The FDA has requested that USWM develop and establish safety information on lofexidine for neonate patient
use. Initial requirements for registration of lofexidine for pediatric use are clearly defined and agreed upon with
the FDA and include a total of five nonclinical studies in animals, seven clinical studies, and commitment to
developing a pediatric specific formulation. The overall goal of this project is to develop a strategic alliance with
NIDA and leverage this partnership to accelerate the development of the pediatric lofexidine program, with
specific emphasis on the activities relevant for NOWS development. The research strategy in this proposal
specifically targets the FDA requested development of lofexidine for NOWS treatment and will be supported by:
1) the development, scale up, and clinical trial material production of a neonatal-appropriate lofexidine
formulation (Specific Aims 1 and 5), 2) completion of pivotal nonclinical studies to establish safety margins in
nonclinical juvenile rodent models considered appropriate to assess safety related to early brain development
and general toxicity (Specific Aims 2, 3, and 4), and 3) completion of a comparative bioavailability study with
adult and pediatric formulations in normal healthy adult volunteers (Specific Aim 6).

## Key facts

- **NIH application ID:** 9960518
- **Project number:** 5R01DA047690-03
- **Recipient organization:** US WORLDMEDS, LLC
- **Principal Investigator:** Kristen Leann Gullo
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $800,000
- **Award type:** 5
- **Project period:** 2018-09-30 → 2021-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9960518

## Citation

> US National Institutes of Health, RePORTER application 9960518, Accelerated Development of Lofexidine for Neonatal Opioid Withdrawal Syndrome (5R01DA047690-03). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/9960518. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*