Danish pharmaceutical company Ascendis Pharma A/S has received news of the US Food & Drug Administration (FDA) declining to approve its experimental therapy, TransCon PTH, which is used to treat adult patients with hypoparathyroidism. The FDA has cited concerns surrounding the manufacturing control of the drug and device combination, but it did not express any concerns about the clinical data that was submitted. This news has caused TD Cowen analyst Yaron Werber to raise concerns about the lack of clarity around the FDA's decision.
Werber has stated that despite the company's US-listed shares rising by 12% in premarket trade, if the FDA were to seek another pre-clinical or clinical study, this could cause approval to be delayed by 1.5-2 years. This would cause the stock to slump, and it would not be a favourable outcome for the company. Currently, Ascendis Pharma A/S has not announced any further plans for the development of TransCon PTH.
The company had been expecting the FDA to approve its hormone disorder therapy after submitting its application for approval. However, in early September, the FDA identified unspecified deficiencies in the application for the therapy. This led to the company waiting for a complete response letter from the FDA, which they received on Monday, declining approval of the therapy.
It is not uncommon for the FDA to decline approval for experimental therapies. The FDA's role is to ensure that drugs and devices that are approved are both safe and effective for patients to use. The FDA has a rigorous evaluation process that pharmaceutical companies must follow before approval can be granted.
Ascendis Pharma A/S has not yet announced any plans to address the FDA's concerns about the manufacturing control of the drug and device combination, and it remains to be seen what the future holds for TransCon PTH. The company's shares will likely be volatile in the short term, given the uncertainty surrounding the FDA's decision.