Pfizer gets EU approval for long-acting human growth hormone, softening blow of FDA rejection

Pfizer and Opko Health have bounced back from a FDA rejection to win approval for long-acting human growth hormone Ngenla in the EU, putting the pair hot on the heels of their rival Ascendis Pharma. 

Ascendis landed EU marketing authorization for once-weekly subcutaneous hormone injection TransCon hGH in January. Pfizer and Opko received EU approval for their once-weekly challenger to Ascendis’ drug around one month later. The near-simultaneous approvals position the companies to fight toe-to-toe for market share in EU countries in the treatment of growth disturbance in kids three years of age and up. 

There are reasons to think Ascendis can compete against its larger rival. When the companies dropped phase 3 data in 2019, Ascendis made the better impression, with its candidate improving on the existing daily injection in terms of height gain, while Pfizer’s rival only demonstrated non-inferiority.

Failing to match Ascendis’ result was a blow to a drug that Pfizer paid $295 million upfront for in 2014. Pfizer forged ahead regardless, filing for approval on both sides of the Atlantic as it sought to protect the growth hormone franchise it built up with the once-daily Genotropin. Now, with the European approval, Pfizer has a shot at retaining patients even as they switch to once-weekly options.

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The situation in the EU contrasts sharply with the market in the U.S., where Ascendis won approval for its once-weekly treatment, sold as Skytrofa, but Pfizer is yet to get the green light. Pfizer was following close behind Ascendis until a request for longer-term data and then a complete response letter set it back.

With Pfizer spinning its wheels, Ascendis has a chance to become the dominant force in the once-weekly market. Ascendis is talking to commercial payers in a bid to broaden access to Skytrofa this year.