In quest to eliminate tropical diseases, Merck KGaA will submit anti-worming drug for approval in young children
As Merck KGaA makes moves to treat the neglected tropical disease schistosomiasis, it has announced that its treatment arpraziquantel has yielded positive Phase III trial results in children between the ages of 3 months and 6 years old, and the company will now seek regulatory approval.
A pediatric version of the standard drug praziquantel, arpraziquantel is an anti-worm medication that prevents newly hatched, parasitic worms from growing or multiplying inside a patient. The trial could offer a cure for millions.
In trials in Cote d’Ivoire and Kenya, more than 90% of participants had no more parasite eggs in their stool or urine after up to three weeks of treatment, Merck KGaA said. The company will apply for a scientific opinion from EMA through the EU’s high-priority medicine procedure, intended for use outside of Europe. Merck KGaA has gotten support from the Global Health Innovative Technology Fund, Farmanguinhos in Brazil and Universal Corporation in Kenya to extend local production capacities.
“With this milestone, we continue our commitment to eliminating schistosomiasis and ensuring all people affected by this neglected tropical disease have access to a life-saving therapy. Together with our consortium partners, we are steadfast in our vision to bring new hope to the world’s most vulnerable populations,” Merck KGaA’s healthcare CEO Peter Guenter said in a press release.
The company is addressing key requirements of the WHO’s 2021-2030 roadmap for neglected tropical diseases through its Schistosomiasis Elimination Program.
The primary endpoint of the trial was defined as no parasite eggs in stool 17 to 21 days after treatment, or in urine 35 to 40 days after treatment. No new potential risks or safety concerns were identified, the company said in a press release.
Arpraziquantel is delivered through an oral tablet. There are an estimated 50 million preschool-aged children with schistosomiasis.
Kio Yamabe, the acting CEO of the GHIT Fund, said in a statement:
Having partnered with the Pediatric Praziquantel Consortium since 2013, we believe that international collaborations like this are key to addressing the burden of major infectious diseases in the developing world. The successful joint development of praziquantel by Consortium partners, Astellas, Merck and Farmanguinhos embodies our unwavering commitment to drive Japanese innovation and technology through global partnerships.
The disease is also known as bilharzia. Famously, Israeli professional cyclist Chris Froome has battled the disease since 2010, and tested positive for another flare-up earlier this year. Doctors told him that the worm could have been dormant for nearly a decade before flaring up again. Froome learned of the flare-up after having digestive issues after the Tour de France.
The drug works by causing severe spasms and paralysis of the worms’ muscles. Often, those are passed in the patient’s stool, but sometimes, they are completely destroyed in the bowels, according to the Mayo Clinic.