FDA declines to OK palopegteriparatide for hypoparathyroidism over manufacturing concerns
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Key takeaways:
- The FDA has rejected a new drug application for palopegteriparatide due to manufacturing control strategy concerns.
- The complete response letter did not indicate concerns regarding clinical trial data.
The FDA has rejected a new drug application for an agent to treat adults with hypoparathyroidism due to concerns with manufacturing control strategy, according to an industry press release.
The FDA issued a complete response letter for palopegteriparatide (TransCon PTH; Ascendis Pharma), an investigational parathyroid hormone prodrug for adults with hypoparathyroidism. According to the press release, the FDA rejected the new drug application due to concerns related to the manufacturing control strategy for variability of the delivered dose in the agent’s drug/device combination product. There were no concerns about the clinical data submitted in the application and no new preclinical studies or phase 3 clinical trials were requested, according to the release.
According to the release, 145 of 154 clinical trial participants continued treatment with palopegteriparatide for up to 3 years. The drug has been well tolerated in clinical trials, with no discontinuations related to the therapy. New patients continue to be enrolled through the U.S. Expanded Access Program.
“We are committed to working collaboratively with the FDA and, because the agency did not suggest that additional phase 3 studies may be needed to demonstrate the product’s safety and efficacy, we believe we are well prepared to address their concerns,” Jan Mikkelsen, president and CEO of Ascendis Pharma, said in a press release. “People with hypoparathyroidism need new treatment options and we are working with urgency to resolve the FDA’s concerns, with the goal of bringing TransCon PTH to patients in the U.S.”
According to the release, Ascendis will request a Type A meeting with the FDA as soon as possible to determine next steps.
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