Pharmanovia announces submission of Paediatric Investigational Plan (PIP) for elamipretide to the medicines and healthcare product regulatory agency (MHRA)

PRESS RELEASE – FOR TRADE, MEDICAL AND BUSINESS MEDIA ONLY

• BARTH SYNDROME IS AN RARE DISEASE WITH LIMITED TREATMENT OPTIONS
• THIS IS THE FIRST STEP IN OUR REGULATORY PATHWAY BEFORE SEEKING FULL MARKET AUTHORISATION IN PATIENTS WITH BARTH SYNDROME

BASILDON, UK, 20 December 2024

Pharmanovia, a global pharmaceutical company that commercialises novel medicines and revitalises, extends and expands the lifecycle of established medicines, today announced the submission of its Paediatric Investigational Plan (PIP) to the MHRA for elamipretide, an investigational treatment for Barth syndrome.

The PIP is an essential regulatory step required under UK law for the development of medicines intended for use in children.  This submission marks a significant milestone in Pharmanovia’s efforts to address the unmet needs of those living with Barth syndrome who could benefit from this treatment.

“This paediatric investigation plan submission to the MHRA reflects our ongoing commitment to addressing unique needs and is our first regulatory step on our journey to bringing this medicine to market. We look forward to continuing our collaboration with regulators, healthcare providers, and families to bring this potential new therapy to those patients living with Barth syndrome,” said Stephen Deacon, Chief Scientific Officer, Pharmanovia.

-Ends-

• Notes to editors
• For further information, please contact:
• Alison Dyson, Director of Communications, Pharmanovia
• 07912887250/ communications@pharmanovia.com
• Or pharmanovia@67health.co.uk

About Pharmanovia

Pharmanovia is a global lifecycle management healthcare company. Our purpose is to make medicines fit for tomorrow, to improve the lives of patients globally.

We do this by rediscovering, repurposing or re-engineering established medicines or by bringing to market novel medicines to improve patient outcomes and experiences.

With a diverse and growing team in over 160 countries across the globe, we deliver high-quality solutions, ethically and sustainably, across our four core therapeutic areas – Endocrinology, Neurology, Cardiovascular and Oncology both in rare and established diseases or conditions.

About elamipretide

Elamipretide is an investigational mitochondrial protective agent that has been shown to normalise mitochondrial structure and function and improve cell viability and organ function across a spectrum of disease models, including models of cardiovascular, renal, metabolic, skeletal muscle, neurodegenerative, and genetic mitochondrial disease. Elamipretide readily penetrates cell membranes and transiently localises to the inner membrane of the mitochondria, where it interacts with cardiolipin, which is known to be depleted in Barth syndrome, and monolysocardiolipin, which is known to be elevated in Barth syndrome.

About Barth syndrome

Barth syndrome is an ultra-rare disease. It is estimated that 1 in 300,000-400,000 people are born with Barth syndrome, but evidence of misdiagnosis is growing, with estimates this figure could be as high as 1 in 140,000. It is an X-linked, multi-organ system disorder characterised by cardiolipin deficiency, cardiomyopathy, musculoskeletal weakness, neutropenia, debilitating fatigue, growth delay, and hypoglycaemia, along with clinical manifestations common in inborn errors of metabolism. The life-threatening disease most commonly affects males but has been reported in females and is associated with genetic mutations in the tafazzin gene causing abnormal cardiolipin remodelling and impaired mitochondrial structure and function.¹

References

^I Clarke et al., 2013. Barth syndrome. Orphanet journal of rare diseases, 8(1), 1-17. https://doi.org/10.1186/1750-1172-8-23