(Reuters) – Ascendis Pharma A/S said on Monday the U.S. Food & Drug Administration had declined to approve the Danish drugmaker’s experimental therapy to treat adult patients with a hormone disorder called hypoparathyroidism, citing concerns linked to manufacturing control of the drug and device combination.
The company’s U.S.-listed shares surged 19% in premarket trade as the agency did not express concerns about the clinical data submitted and did not seek fresh pre-clinical or late-stage trials in a so-called complete response letter.
Ascendis said it would request a meeting with the FDA as soon as possible on the best path forward.
Shares have lost about 35% of their value through Friday’s close of $69.96 since the FDA early last month identified unspecified deficiencies in the company’s application for approval of the therapy, TransCon PTH.
In a note on Friday, TD Cowen analyst Yaron Werber raised concerns about the lack of clarity around the FDA decision following the Ascendis’ post-earnings conference late Thursday.
He said the stock would slump if the FDA sought another pre-clinical or clinical study as this would delay approval by 1.5-2 years. He expected the stock to advance if the delay was to the end of this year.
Werber has estimated peak sales of 827 million euros ($911 million) for the therapy in case of an approval this year, but anticipates it crossing 1 billion euros if the uptake is strong.
TransCon PTH is being developed as a once-daily therapy to replace the deficient or absent parathyroid hormone, a characteristic feature of the disease that affects over 200,000 people worldwide, according to the company’s website.
($1 = 0.9079 euros)
(Reporting by Mariam Sunny in Bengaluru; Editing by Anil D’Silva and Sriraj Kalluvila)
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