The U.S. Food & Drug Administration (FDA) on Tuesday extended review for Ascendis Pharma A/S‘ (NASDAQ:ASND) New Drug Application (NDA) for TransCon CNP (navepegritide) for children with achondroplasia.
• ASND is in positive territory. Check the fundamentals here.
Achondroplasia is a genetic disorder causing dwarfism, characterized by short limbs, a normal-sized torso, and a large head.
It is the most common form of skeletal dysplasia and results from a mutation in the FGFR3 gene, which impairs cartilage from turning into bone, particularly in the long bones of the arms and legs.
The FDA said the information submitted on Nov. 5, 2025, related to the post-marketing requirement constituted a major amendment to the NDA. Accordingly, the FDA has extended the PDUFA target action date by three months to Feb. 28, 2026.
“We have responded to all outstanding requests from the FDA, including the request for a revised protocol for the post-marketing study, which we received as the lone item for discussion at our late-cycle meeting,” said Jan Mikkelsen, President and CEO at Ascendis Pharma.
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In November, Ascendis Pharma announced that pivotal week 52 results from its ApproaCH Trial of investigational once-weekly TransCon CNP (navepegritide) in children with achondroplasia have been published in JAMA Pediatrics, a journal of the American Medical Association.
The authors report that treatment with TransCon CNP led to significantly higher annualized growth velocity (AGV) at week 52 compared to placebo (primary endpoint), as well as improved lower-limb alignment and body proportionality and positive changes in health-related quality of life, with a safety and tolerability profile similar to placebo.
Earlier in November, the company shared a new pooled analysis of TransCon PTH (palopegteriparatide) in adults with hypoparathyroidism.
The data showed sustained and clinically meaningful improvements in renal function through year 3 of Phase 2 PaTH Forward and Phase 3 PaTHway trials.
At Year 3, ≥91% of patients receiving palopegteriparatide in both trials were independent from conventional therapy, and ≥84% patients had normocalcemia.
Sustained and clinically meaningful improvements in eGFR (≥ 5 mL/ min / 1.73 m2) were observed in 70.3%, with numerically greater improvements observed in those with lower baseline eGFR.
ASND Price Action: Ascendis Pharma stock is up 1.44% at $213.03 at publication on Friday.
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