Critical Path Global Impact Conference highlights collaborative progress 

The Critical Path Global Impact Conference brought together patient advocates, regulators, and industry leaders to engage in vital conversations about improving drug development and approvals. This gathering underscored the importance of collaboration and breaking down silos in data collection, ensuring that every piece of data can serve as a meaningful outcome measure in advancing research for rare diseases. Participants across several portions of the health sector focused on a common mission: accelerating drug development. 

FDA hearing highlights critical debate over Obeticholic Acid for PBC treatment

In a disappointing announcement for #PBC patients, on September 13 the FDA Gastrointestinal Drugs Advisory Committee (GIDAC) did not recommend continued marketing authorization for Intercept Pharmaceuticals’ obeticholic acid (OCA), a decision that has serious implications for patients who have relied on this treatment since its accelerated approval in 2016. This outcome means that many may now face the loss of an important option for managing their condition.

While we respect the GIDAC’s decision process and recognize their responsibility to ensure patient safety, it is still a disappointing result for the #rare #liver disease community. Intercept Pharmaceuticals, alongside patient advocates, presented extensive real-world data highlighting OCA’s continued benefits. Many patients, including Global Liver Institute Council members L. Marie Asad and Dr. Cecilia Duenas, shared their personal stories, stressing the importance of keeping this treatment available. Despite this powerful testimony, the GIDAC did not recommend moving this molecule forward as a second-line therapy for patients to consider in their care. 

Moving forward, the FDA’s accelerated approval process remains critical to ensure our liver patients can receive timely treatment when there are limited options, while clinical trials continue to collect evidence for full FDA approval.

Global Liver Institute remains committed to advocating for patients and will continue working to ensure that their needs and voices are prioritized.

Celebrating advancements in cholestatic disease treatments in the EU 

Ipsen has achieved progress in the fight against cholestatic diseases, marking two important victories for patients. The conditional approval of Iqirvo® (elafibranor) in the EU introduces a much-needed treatment option for PBC patients, expanding choices in a field with limited alternatives. In the same week, Kayfanda® (odevixibat) was granted approval under exceptional circumstances for cholestatic pruritus in Alagille syndrome (ALGS) to address pruritus, a severe symptom that significantly affects patient quality of life.

Orphan designation granted for a treatment for polycystic liver disease  

Camurus’s investigational medicinal product, octreotide subcutaneous (SC) depot (CAM2029) for polycystic liver disease (PLD) has received orphan designation from the EMA. This important designation comes at a time when, lacking treatment options, PLD results in patients requiring liver transplants in severe cases. CAM2029 is an injectable formulation of octreotide that may be suitable for home administration. Octreotide is a hormone recognized for its ability to regulate other hormones and inhibit tumor growth. This therapy is designed not only to mitigate disease progression but also to improve symptom management, which is critical for enhancing patient quality of life. Clinical trials for this drug are currently enrolling patients.