Ascendis to present infant achondroplasia data at ESPE 2026

Ascendis Pharma will share sentinel cohort data from its reACHin pivotal trial of navepegritide in infants under two at ESPE 2026.

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A brightly lit, clean medical examination room features a large CT scanner with blue brain and neuron illustrations on its glowing gantry, a computer monitor on a rolling cart, a medical bench, and a window.

Ascendis Pharma has announced it will present data from its Endocrinology Rare Disease portfolio at ESPE 2026, the annual congress of the European Society for Paediatric Endocrinology, held in Marseille from 8 to 10 September. The centrepiece is a podium presentation of the first sentinel cohort data from the reACHin trial, a pivotal study of TransCon CNP (navepegritide) in infants with achondroplasia aged under two years.

Achondroplasia is the most common form of short-limbed dwarfism, caused by a gain-of-function mutation in the FGFR3 gene that inhibits endochondral bone growth. Clinical interest in treating infants at the earliest possible stage has grown significantly, given that skeletal and neurological complications, including foramen magnum stenosis, can manifest within the first years of life. Ascendis is positioning early intervention with navepegritide as a strategy to reduce those neuromusculoskeletal complications before irreversible changes occur.

The ESPE programme

The reACHin sentinel data will be presented on Tuesday 8 September by Genevieve Baujat, M.D., in the Free Communications session on Growth and Syndromes. Sentinel cohorts typically enrol a small number of subjects at the outset of a pivotal trial to confirm safety and dosing before broader enrolment proceeds; the data will therefore be read as an early safety and tolerability signal rather than an efficacy readout.

Beyond achondroplasia, Ascendis is showing two hypoparathyroidism posters focused on adolescent patient-reported outcomes, and two presentations covering its lonapegsomatropin programme in children with short stature due to Turner syndrome, SHOX deficiency, small for gestational age status, or idiopathic short stature.

Market context and competitive landscape

Navepegritide is a long-acting C-type natriuretic peptide prodrug delivered via the company's TransCon platform, which uses transient conjugation to extend drug half-life. The mechanism is distinct from that of vosoritide (BioMarin's Voxzogo), a daily subcutaneous injection of a CNP analogue approved in Europe and the United States for achondroplasia in children aged two and above. BioMarin has also been exploring extension of vosoritide into younger age groups, making the infant indication a contested frontier. Should Ascendis demonstrate a clean safety profile in the reACHin sentinel cohort, it will strengthen the case for a once-weekly or less-frequent dosing schedule as a differentiator for families with young children.

Aimee Shu, M.D., Executive Vice President and Chief Medical Officer at Ascendis Pharma, said the company was particularly eager to share "these early data from our trial of TransCon CNP in infants with achondroplasia," adding that early intervention "may help mitigate the neuromusculoskeletal complications and co-morbidities associated with this condition."

The broader paediatric rare endocrine disease space is drawing increasing venture and large-pharma attention, partly because orphan designations and paediatric rare disease priority review vouchers improve the commercial calculus for small-population programmes. Ascendis has approved revenues from its hypoparathyroidism franchise, giving it a commercial base from which to fund continued pipeline expansion. Investors will look to the full reACHin dataset, including longer-term growth velocity and safety data across a larger cohort, as the next material readout.

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