Target product profile for sleeping sickness

Sleeping sickness

Target product profile for sleeping sickness

Target product profile 1: A new treatment for stages 1 and 2 disease

DNDi aims to develop an oral or intramuscular treatment for sleeping sickness (human African trypanosomiasis), maximum one week in duration, that is effective against T.b. gambiense and rhodesiense parasites in all patients in both stages of the disease.

	Ideal	Acceptable
Target population	Effective against stages 1 and 2 Effective in melarsoprol refractory patients All patients, including pregnant and lactating women	Effective against stages 1 and 2
Target species	Efficacy against both T.b. gambiense and T.b. rhodesiense	Efficacy against T.b. gambiense only
Efficacy	Clinical efficacy > 95% at 18-month follow-up	Clinical efficacy > 95% at 18-month follow-up
Safety/tolerability	<0.1% drug related mortality No monitoring for adverse events (AEs)	<1% possibly related mortality Weekly simple lab testing (field testing) for AEs
Formulation	Adult and paediatric formulations
Treatment regimen	<7 days oral once daily or <7 days intramuscular injection once daily	10 days oral or <10 days intramuscular injection
Stability	Stability in climatic zone 4 for >3 years	Stability in climatic zone 4 for > 12 months
Cost	< EUR 30 / course (drug cost only)	< EUR 100 / course < EUR 200 / course OK if very good on other criteria

Target product profile 2: A treatment to be developed only if a newly developed second-stage treatment fails to show efficacy and is already in advanced clinical trials

DNDi aims to develop a three-day, oral or intramuscular treatment for sleeping sickness (human African trypanosomiasis) that is highly effective against T.b. gambiense and rhodesiense parasites in all stage-1 patients at a very low cost and with an impeccable safety profile.

	Ideal	Acceptable
Target population	Effective against stage 1 Suitable for pregnant and breastfeeding women, and for children	Effective against stage 1 Suitable for pregnant and breastfeeding women, and for children
Target species	Broad spectrum (T.b. gambiense and rhodesiense)	Efficacy against T.b. gambiense only
Efficacy	Clinical efficacy >95% at 18-month follow-up	Clinical efficacy no worse than pentamidine
Safety/tolerability	0% drug related mortality No monitoring for AEs*	0% drug-related mortality No monitoring for AEs
Formulation	Adult and paediatric formulations	Adult and paediatric formulations
Treatment regimen	<3 days treatment single dose oral or intramuscular injection (single dose in animal models, long t1/2)	<7 days treatment 2-3 daily doses oral or intramuscular injection
Stability	Stability in climatic zone 4 for > 4 years	Stability in climatic zone 4 for >2 years
Cost	<EUR 10 / course	<EUR 30 / course

* No specific monitoring needed

Making medical history for neglected patients

We develop urgently needed treatments for neglected patients and ensure they’re affordable, available, and adapted to the communities who need them

Young boy sitting on a hospital bed being examined by a nurse

Chagas disease

Causes heart and vital organ damage, after people are bitten by blood-sucking bugs

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Cryptococcal meningitis

Without treatment, deadly for thousands of people with advanced HIV

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Cutaneous leishmaniasis

Leaves disfiguring, life-long scars that lead to severe social stigma

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Doctor with young patient in hospital setting

Dengue

Rapidly spreading climate-sensitive disease with no specific treatment

We’re building a global partnership with dengue-endemic countries to develop a first treatment

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Filaria: river blindness

Lead to unbearable itching, disfiguring skin lesions, and even blindness

We’re working to develop a safe, effective, and affordable drug for the prevention and treatment of this debilitating disease

Hepatitis C

Millions are left without treatment even though effective drugs exist

We’ve delivered a treatment as simple, safe, and effective as the best drugs available today – at a fraction of the cost

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Mycetoma

Often ends in amputation, after people get infected from stepping on a thorn

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Paediatric HIV

Without treatment, half of children die before their second birthday

We’ve developed a strawberry-flavoured treatment to meet the needs of children long neglected by the global HIV response

Pandemic Preparedness

The COVID-19 pandemic intensified global health inequalities

We’re bringing together partners and accelerating research to prepare for future viral pandemics in low-resource settings