U.S. FDA declines to approve Pfizer-OPKO growth hormone deficiency treatment

(Reuters) – Pfizer Inc (PFE.N) said on Friday the U.S. health regulator has declined to approve the treatment for growth hormone deficiency in children that it developed with partner OPKO Health Inc (OPK.O).

Pfizer’s statement did not mention a reason for the U.S. Food and Drug Administration’s (FDA) decision in its complete response letter.

“We will work closely with the FDA to determine the best path forward to bring this important once-weekly treatment option to pediatric growth hormone deficiency patients and their families,” said Brenda Cooperstone, chief development officer of Rare Disease, Pfizer Global Product Development.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo

Shares of Pfizer were down 0.4% in aftermarket trading, while OPKO dropped 9.2%.

The application was based on results from a late-stage study which showed that treatment with somatrogon once a week was non-inferior to Pfizer’s approved growth hormone, Genotropin, which is to be administered daily.

An approval would have allowed Pfizer to compete with Ascendis Pharma (A71.F), whose once-weekly growth hormone injectable was approved by the FDA in August and launched in October.

Pfizer has submitted applications to regulators of various countries and Japan earlier this week approved the drug, which will be sold under the brand name Ngenla.

Growth hormone deficiency is a rare disorder where the pituitary gland does not produce enough growth hormone to stimulate the body to grow. It is characterized by abnormally short height and affects one in about 4,000 to 10,000 children globally.

Reporting by Leroy Leo and Mrinalika Roy in Bengaluru; Editing by Devika Syamnath

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