London, UK – 01/07/2024: - ADVANZ PHARMA Holdco Limited (“ADVANZ PHARMA” or “the Company”), a UK headquartered pharmaceutical company, acknowledges that the Committee for Medicinal Products for Human Use (“CHMP”) has recommended the European Commission should revoke the EU conditional marketing authorisation of OCALIVA® for 2nd line use in patients with the rare disease Primary Biliary Cholangitis (PBC).
The CHMP recommendation is the outcome of an Article 20 referral initiated by European Medicines Agency (“EMA”) in October 2023 to reassess the benefit-risk of OCALIVA® in PBC. The OCALIVA® conditional marketing authorization for Europe was granted in 2016 with a requirement to demonstrate improvement in clinical outcomes through additional clinical studies, specifically, randomized placebo-controlled studies in an advanced stage patient population. Following recruitment challenges and patients moving from placebo to commercially available medicines, the required studies were prematurely terminated in 2021 upon the recommendation of an independent data monitoring committee and in agreement with the EMA, precipitating the Article 20 referral.
“We are very disappointed with this recommendation”, said Steffen Wagner, CEO at ADVANZ PHARMA. “Based on the extensive safety and real-world efficacy data that we – together with many others in the academic expert community – have generated, we consider OCALIVA® to have a positive benefit-risk balance in patients with PBC. This view is supported by many experts, physicians, patient representatives and scientific societies for hepatology across Europe. We are determined to help ensure that patients in the EU benefitting from OCALIVA® are not forgotten, and we are carefully considering next steps.”
The EMA public health communication references the ability for ADVANZ PHARMA to continue – subject to local laws and regulations – to supply OCALIVA® in the EU on a compassionate access or named patient programme basis for existing patients in the event that the European Commission upholds the revocation recommendation. Further information is available from medicalinformation@advanzpharma.com.
The CHMP recommendation will require the adoption by the European Commission. Until the European Commission has reached a final determination on this matter, the current EU conditional marketing authorisation for OCALIVA® remains in place and patients in the EU may continue to receive unchanged access to OCALIVA®.
About OCALIVA® and PBC
Primary biliary cholangitis (PBC) is a rare, serious and progressive autoimmune disease that damages the bile ducts in the liver. OCALIVA® is a film-coated tablet containing obeticholic acid (OCA), a selective and potent agonist of the farnesoid X receptor (FXR) which regulates bile acid, inflammatory, fibrotic and metabolic pathways and decreases intracellular hepatocyte concentrations of bile acids. OCALIVA® is indicated for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.
About ADVANZ PHARMA
ADVANZ PHARMA is a global pharmaceutical company with the purpose to improve patients’ lives by providing and enhancing the specialty, hospital, and rare disease medicines they depend on. Our headquarters are in London, UK. We have commercial sales in more than 90 countries globally and have a direct commercial presence in more than 20 countries, including key countries in Europe, the US, Canada, and Australia, a Centre of Excellence in Mumbai, India, as well as an established global distribution and commercialisation partner network. ADVANZ PHARMA’s product portfolio and pipeline comprises innovative medicines, specialty generics & biosimilars, and originator brands. Our products cover a broad range of therapeutic areas, including hepatology, gastroenterology, anti-infectives, critical care, endocrinology, oncology, CNS, and, more broadly, rare disease medicines. Our ambition is to be a partner of choice for the commercialisation of specialty, hospital, and rare disease medicines in Europe, Canada, and Australia. In line with our ambition, we are partnering with biopharma and development companies to bring medicines to patients. For more information, please visit our Website or LinkedIn.
For media inquiries, please contact:
Fiona Huzarski
ADVANZ PHARMA
Tel: +44 7557 602096
Email: fiona.huzarski@advanzpharma.com
