The National Pharmaceutical Regulatory Agency (NPRA) of Malaysia has granted a conditional registration for a safe, effective hepatitis C treatment developed by a public-private partnership bringing together the Malaysian Ministry of Health, not-for-profit research and development organization Drugs for Neglected Diseases initiative (DNDi), Egyptian pharmaceutical company Pharco, Malaysian pharmaceutical company Pharmaniaga Berhad, and non-governmental-organization Médecins Sans Frontières/Doctors Without Borders (MSF).
This is the very first drug for hepatitis C virus (HCV) to be developed through South-South collaboration and with funding and clinical support from non-profit organizations.
This partnership was formed to address one of the more intractable public health challenges of the past decade: the lack of access to affordable direct-acting antivirals (DAAs), a newer generation of powerful HCV treatments that can cure patients in three to six months. HCV, which can lead to chronic liver disease, cirrhosis, cancer, and death, affects about 58 million people worldwide, but only about 13% have received treatment to date. The disease causes around 300,000 deaths a year.
'Although hepatitis can be cured, there is a vicious circle that stands in the way of providing treatment to all in need: the disease is mostly a 'silent killer', the diagnostic process is complex, so people go unfound, and DAAs are often too expensive,' said Tan Sri Dato' Seri Dr Noor Hisham Abdullah, Director General of Health, Ministry of Health, Malaysia. 'Malaysia decided to act to break this vicious circle. We are actively screening to find 'missing' patients, rolling out simpler diagnostic tests, and ensuring we have access to the best prices for treatments, including by conducting clinical research to identify additional affordable treatment options. Today's announcement is a milestone on Malaysia's long journey to achieve the World Health Organization goal of eliminating hepatitis C by 2030.'
The approval today concerns a new drug, ravidasvir, for the treatment of chronic HCV infection in adults in combination with other medicinal products. Ravidasvir was developed by the partnership for use with sofosbuvir, an existing DAA, as an affordable, simple, and efficacious public health tool.
DNDi and partners conducted STORM-C-1, an open-label clinical trial to assess the efficacy, safety, and pharmacokinetics of ravidasvir combined with sofosbuvir in Malaysia and Thailand. The study was sponsored by the Ministries of Health of Malaysia and Thailand, and DNDi. In results published in April 2021 in The Lancet Gastroenterology & Hepatology, the combination showed cure rates of 97% and was well tolerated in a diverse adult population with chronic HCV infection. This included people infected with genotype 3 of the virus, which is particularly hard to treat. No clinically significant drug-drug interactions with the antiretrovirals commonly used to treat HIV were found, which will make this new combination particularly useful to clinicians.
'The development of this new drug is the result of a partnership between public and private actors sharing the same public health objective from the very start: the development of an affordable medicine. It is a concrete example of how research and development can deliver innovation driven by public health needs, rather than market imperatives,' said Dr Bernard Pécoul, Executive Director of DNDi.
Ravidasvir is an oral NS5A inhibitor discovered and owned by Presidio Pharmaceuticals. It was licensed to Egyptian drug manufacturer Pharco Pharmaceuticals and DNDi for clinical development and commercialization. In an earlier Phase III clinical trial in Egypt conducted by Pharco, ravidasvir + sofosbuvir showed cure rates of up to 100% in patients with genotype 4. Pharco and DNDi partnered with Malaysian drug manufacturer Pharmaniaga for the registration and supply of ravidasvir in Malaysia and South-East Asia.
'This new treatment will be a powerful tool in our arsenal to make the vision of a hepatitis C-free world a reality,' said Dr Sherine Helmy, CEO of Pharco Pharmaceuticals. 'This is why this new combination will be sold at an affordable price between USD 300-500 for a 12-week treatment course.'
'NPRA approval is a huge milestone for all parties who have been working tirelessly to make a safe, effective, accessible and, most importantly, affordable treatment for hepatitis C in Malaysia,' said Datuk Zulkarnain Mohamed Eusope, Pharmaniaga Group Managing Director. 'This is a first for us at Pharmaniaga, a generics company, being involved in the development of a new chemical entity. We thank the NPRA for their continuous support, and for prioritizing the approval, obtained within 15 months, compared to standard timelines of two years or more. We hope this latest development will increase options and facilitate access for the public to obtain a more affordable treatment for HCV.'
The STORM-C project was financed by MSF's Transformational Investment Capacity initiative, with the objective of increasing access to treatment for HCV patients in low- and middle-income countries.
'Hundreds of thousands of people die each year from the consequences of hepatitis C and expanding the access to cure is a medical humanitarian imperative,' said Pierre Mendiharat, Deputy Operations Director for MSF. 'HCV affects vulnerable and often marginalized populations including people who use drugs and people co-infected with HIV. To eliminate HCV, we need easy-to-use, simple, and affordable treatments that work, like the ravidasvir + sofosbuvir combination.'
Plans to register the new ravidasvir + sofosbuvir combination are advancing in other countries, including in Latin America.
'Our goal is now to help foster the political will and financing needed for wide-scale roll-out of lifesaving testing and treatment for hepatitis C globally,' said Dr Bernard Pécoul. 'The Malaysian example is a showcase of what can be done with the right will, the right partners, and the right tools.'
The Lancet Gastroenterology & Hepatology