Pharmanovia and Stealth BioTherapeutics enter new licensing agreement for novel Barth syndrome treatment

Pharmanovia and Stealth BioTherapeutics have announced that they have entered into a new licensing agreement for the marketing and further development of elamipretide as a treatment for Barth syndrome in Europe and the Middle East and North Africa (MENA).
This agreement gives Pharmanovia the licensing rights to the drug in Europe and MENA, however it plans to work with Stealth on the final analyses of the studies demonstrating elamipretide’s potential for this indication. Various clinical trials have already been carried out showing positive results and have demonstrated the drug’s safety and efficacy, as well as its efficacy compared to control groups.
James Burt, Pharmanovia’s CEO, commented: “This partnership is our third new chemical entity in as many months, further broadening our treatment portfolio, as we set our sights on improving the experience and lives of patients globally. […] We are delighted to have been recognised by Stealth BioTherapeutics as the right overseas partner to launch and potentially bring this novel treatment to patients in the EU, Switzerland, Norway, UK, Iceland and MENA. Beyond revitalising and adding value to iconic brands, utilising our extensive technical and commercial platform to address unmet needs with novel medicines that complement our existing portfolio across our core therapeutic areas, in this instance cardiology, is a key strategic priority for us. […] Only 5% or fewer rare diseases are estimated to have an approved treatment option, therefore we’re especially excited to work with authorities and regulators on potentially bringing to market the first specific treatment option for those living with Barth syndrome.“
Reenie McCarthy, Stealth BioTherapeutics’ CEO, added: “We are committed to broadening global access to elamipretide for patients living with Barth syndrome. Partnering with Pharmanovia is a natural choice to achieve that goal given their experience in bringing medicines to patients across Europe and MENA and our mutual alignment on elamipretide’s potential to address the unmet medical needs of patients living with Barth syndrome.”