ADVANZ PHARMA’s response to European Commission revocation

ADVANZ PHARMA’s response to European Commission revocation of conditional marketing authorisation for OCALIVA® in rare disease Primary Biliary Cholangitis (PBC)

OCALIVA® is the only farnesoid X receptor (FXR) agonist for patients with PBC and, until now the only approved and available second-line treatment option in Europe
ADVANZ PHARMA is considering all options to enable continued access for patients

London, UK – 3 September 2024: - ADVANZ PHARMA Holdco Limited (ADVANZ PHARMA or the Company), a UK headquartered pharmaceutical company, today announces that the European Commission (EC) has revoked the conditional marketing authorisation (CMA) of OCALIVA® (obeticholic acid) in Europe for second-line treatment of patients with the rare disease Primary Biliary Cholangitis (PBC).

The EC decision is based on a recommendation in June 2024, from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), to revoke the CMA of OCALIVA® across Europe, following a non-pharmacovigilance Article 20 procedure to reassess the benefit-risk profile of the medicine in PBC. This was not based on any safety concerns.

ADVANZ PHARMA disagrees with the EC’s decision, which leaves thousands of patients with PBC who are responding well to OCALIVA® at increased risk of disease progression with limited treatment options. The Company is currently considering all options to help ensure continued access to OCALIVA®.

Steffen Wagner, CEO at ADVANZ PHARMA, commented: “We strongly disagree with the European Commission’s decision to withdraw the conditional marketing authorisation for OCALIVA®, the only FXR agonist for patients with PBC, and until now the only approved and available second-line treatment option in Europe. The removal of OCALIVA® could have a profoundly negative impact on the lives of the thousands of patients with PBC across Europe who have benefitted from this important treatment over many years. The decision puts them at increased risk of disease progression, including serious liver harm, liver transplantation or death.

“We are committed to supporting patients with PBC and will consider all available potential routes to maintain availability of this important treatment option. We will continue to work with the relevant authorities across the EU to help ensure OCALIVA® remains available for patients who require it.”

The Company maintains that the CHMP recommendation did not adequately consider the totality of available data supporting the efficacy and safety of OCALIVA® in PBC, in particular the wealth of positive real-world evidence (RWE) gathered from more than seven years of clinical use representing over 47,000 patient-years of treatment experience. Instead, it largely relied on one analysis based on a single, randomised placebo-controlled trial, Study 747-302 (COBALT), which had multiple limitations, including the fact that patients in the placebo arm unsurprisingly chose to switch to commercially available therapy, but were required to be analysed as placebo treated patients under the Intention to Treat (ITT) methodology.

The findings from the COBALT study, recently published in the American Journal of Gastroenterology¹, highlight the challenges of conducting long term outcomes studies in rare diseases when therapies are commercially available.

The patient community, leading experts, physicians, and hepatology and gastroenterology scientific societies, have all raised concerns to the EC that the removal of OCALIVA® will leave patients without an important treatment option for this life-threatening rare liver disease. All major scientific societies recommend OCALIVA® as a second-line treatment option in their clinical guidelines.

The EMA has referenced 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. ADVANZ PHARMA is committed to supporting patients and will ensure supplies are available depending on national competent authority approved provisions.

OCALIVA®, conditionally approved by the EMA in December 2016, was the only approved and available second-line treatment option for patients with PBC in Europe. As the only approved FXR agonist, OCALIVA® has a different mechanism of action from other treatments, making it an important option for patients with PBC, a condition where treatment modalities are already very limited.

Patients should consult their treating physician if they have any questions.

Further information is available from medicalinformation@advanzpharma.com.

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 PBC 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 enquiries, please contact:
ADVANZ PHARMA
Courtney Baines
Email: courtney.baines@advanzpharma.com

ICR Consilium
Email: advanzpharma@icrhealthcare.com

GL/OCA/NP/0056
Date of preparation: September 2024

¹ https://journals.lww.com/ajg/abstract/9900/cobalt__a_confirmatory_trial_of_obeticholic_acid.1290.aspx

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ADVANZ PHARMA’s response to European Commission revocation of conditional marketing authorisation for OCALIVA® in rare disease Primary Biliary Cholangitis (PBC)

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