Ascendis Pharma Charts Course for EU Approval of TransCon CNP, Offering New Hope for Achondroplasia

Ascendis Pharma has taken a monumental step towards transforming the lives of individuals with achondroplasia in Europe. The company recently announced the submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for TransCon CNP, an innovative investigational treatment designed to address the underlying causes of this rare genetic disorder.

Achondroplasia is the most common form of skeletal dysplasia, a condition characterized by disproportionate short stature and other skeletal abnormalities, stemming from a genetic mutation that impacts bone growth.

For years, patients and their families have sought effective treatments beyond symptomatic management. TransCon CNP, a long-acting C-type natriuretic peptide (CNP) analog, represents a potential paradigm shift by targeting the pathophysiology of achondroplasia.

The comprehensive MAA submitted to the EMA is underpinned by a robust body of clinical evidence.

This includes compelling data from the global ACcomplisH trial, which encompasses both its Phase 2 and subsequent Phase 3 extension phases, as well as crucial insights from the ACcomplisH EU trial. These studies have rigorously evaluated the safety and efficacy of TransCon CNP, demonstrating its potential to positively impact growth and other aspects of health in children with achondroplasia.

This significant European regulatory milestone follows closely on the heels of similar progress in the United States.

In April, the U.S. Food and Drug Administration (FDA) accepted Ascendis Pharma's Biologics License Application (BLA) for TransCon CNP, setting a Prescription Drug User Fee Act (PDUFA) date of October 21, 2024. The simultaneous advancement in both major regulatory regions underscores the company's commitment to making this therapy accessible worldwide.

The potential of TransCon CNP has also been recognized through key regulatory designations.

It has been granted Breakthrough Therapy designation by the FDA in the U.S., a status reserved for treatments that may offer substantial improvement over available therapies for serious conditions. Additionally, TransCon CNP holds Orphan Drug designation in both the U.S. and the EU, highlighting its importance for a rare disease population with unmet medical needs.

With these submissions, Ascendis Pharma moves closer to potentially offering a new, much-needed therapeutic option that could significantly improve the quality of life and long-term health outcomes for children and families affected by achondroplasia in Europe and beyond.

The scientific community and patient advocates eagerly await the outcomes of these regulatory reviews, holding high hopes for this promising treatment.