New Oral Treatment for Sleeping Sickness Receives Positive Scientific Opinion from EMA

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Utilize (CHMP) has issued a positive scientific opinion for acoziborole Winthrop, a single-dose oral treatment for human African trypanosomiasis (HAT), also known as sleeping sickness, caused by Trypanosoma brucei gambiense (g-HAT). This opinion is intended to facilitate access to the medication in low- and middle-income countries where g-HAT is endemic, operating under the EU-M4all program Sanofi.

Understanding Sleeping Sickness

Human African trypanosomiasis is a neglected tropical disease spread by the bite of infected tsetse flies Sanofi. The World Health Organization (WHO) has set a goal to eliminate transmission of the gambiense strain of the disease by 2030 Sanofi. Without treatment, the disease is fatal. Initial symptoms include fever, headache, and rash, progressing to neurological symptoms like sleep disturbances and behavioral changes as the parasite invades the central nervous system Sanofi.

Acoziborole: A Potential Game-Changer

Acoziborole Winthrop is indicated for both first-stage and second-stage (including severe cases) g-HAT in adults and adolescents weighing at least 40 kg Sanofi. The drug is believed to operate by blocking an enzyme essential for the parasite’s protein production, ultimately leading to its death.

Current treatments for g-HAT are complex, often requiring multiple doses, intravenous infusions, or intramuscular injections, and frequently necessitate hospitalization. Diagnosis typically involves a lumbar puncture to determine the disease stage NCBI. Acoziborole Winthrop offers a simpler approach: a single oral dose effective against all stages of the disease, eliminating the need for lumbar puncture and hospitalization Sanofi.

Clinical Trial Results

A Phase II/III clinical trial conducted in the Democratic Republic of Congo and Guinea demonstrated promising results. The study, involving 208 adults and adolescents, showed a treatment success rate of approximately 95% (159 out of 167) in patients with second-stage disease and 100% (41 out of 41) in those with first- and intermediate-stage g-HAT after 18 months Sanofi. Treatment success was defined by the absence of clinical signs, undetectable parasites, and a normal white blood cell count.

Safety and Side Effects

Common side effects reported during trials included headache, fever, weakness, decreased appetite, and tremor Sanofi. Acoziborole Winthrop is not recommended for individuals with pre-existing heart rhythm problems or those taking certain HIV and anti-infection medications. Patients will receive a card detailing these risks. A post-authorization study will monitor the drug’s real-world safety for three months after treatment.

Next Steps and Global Impact

The EMA’s positive scientific opinion will facilitate the WHO prequalification process and national registration of Acoziborole Winthrop in endemic countries, streamlining its use in public health programs. The application received an accelerated review to expedite access for patients. The EU-M4all program, in collaboration with the WHO and target countries, combines EMA’s scientific expertise with local disease knowledge to address public health needs Sanofi.