Benzinga | Jan 20, 2022 08:12AM EST

Japan's Ministry Of Health, Labour And Welfare Approves Pfizer And OPKO's NGENLA (Somatrogon), A New Long-Acting Treatment For Pediatric Growth Hormone Deficiency

https://www.globenewswire.com/news-release/2022/01/20/2370129/0/en/Japan-s-MHLW-Approves-Pfizer-and-OPKO-s-NGENLA-Somatrogon-a-New-Long-Acting-Treatment-for-Pediatric-Growth-Hormone-Deficiency.html

Pfizer Japan Inc. and OPKO Health, Inc. (NASDAQ:OPK) announced today that the next generation long-acting growth hormone injection, NGENLA(r) (somatrogon) Inj. 24 mg Pens and 60 mg Pens, has been approved by the Ministry of Health, Labour and Welfare (MHLW) in Japan. NGENLA(r) is a once-weekly long-acting recombinant human growth hormone, for the indication of short statue due to growth hormone deficiency without closed epiphyses. NGENLA(r) provides patients with pediatric growth hormone deficiency (GHD) with a new option that reduces treatment frequency from daily injections to once-weekly injections.

This approval is based on the results of a Phase 3 study conducted in Japanese subjects and a global Phase 3 clinical study, both of which were conducted in subjects with pediatric GHD, and both of which compared the efficacy and safety of once-weekly NGENLA(r) with GENOTROPIN(r) (somatropin), a recombinant human growth hormone for injection administered once-daily. In both studies, NGENLA(r) showed comparable efficacy to GENOTROPIN in the primary endpoint of annual height velocity at 12 months. NGENLA(r) was generally well tolerated in both studies, with comparable safety to that of GENOTROPIN administered once-daily with respect to the types, numbers and severity of the adverse events observed between the treatment arms.

"We are pleased to receive approval for once-weekly NGENLA(r), which offers a new treatment option for pediatric GHD patients that can help reduce the burden associated with daily growth hormone administration. We wish to express our gratitude to the patients and their families who participated in the clinical studies and to all the sites conducting these trials," said Taro Ishibashi, President of Pfizer R&D Japan G.K.

In 2014, Pfizer and OPKO Health entered into a worldwide agreement for the development and commercialization of somatrogon for the treatment of GHD. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing somatrogon for GHD.

About NGENLA

Product name NGENLA(R) Inj.24mg Pens NGENLA(R) Inj.60mg PensGeneral name Somatrogon (recombination)INDICATIONS Short stature due to growth hormone deficiency without closed epiphysesDOSAGE AND Generally, Somatrogon (recombination) 0.66 mg per kilogram bodyADMINISTRATION weight is administered once-weekly by subcutaneous injection.MarketingAuthorization Pfizer Japan Inc.Holder

About the Japan Phase 3 Study

The Phase 3 study of NGENLA(r) in 44 treatment-na?ve Japanese pre-pubertal children with pediatric GHD was a 12-month, open-label, randomized, active-controlled, parallel-group study of the efficacy and safety of weekly NGENLA(r) compared to recombinant human growth hormone (r-hGH), GENOTROPIN (somatropin) for injection treatment administered once-daily. Eligible patients were randomized in a 1:1 ratio to receive either once-weekly NGENLA(r) or GENOTROPIN administered once-daily (reference therapy, 0.025 mg/kg/day which is equivalent to 0.175 mg/kg/week). To obtain pharmacokinetic information of three different weekly doses in Japanese pediatric GHD patients, NGENLA(r) treated patients received 0.25 mg/kg/week for 2 weeks, followed by 0.48 mg/kg/week for 2 weeks followed by 0.66 mg/kg/week for the remaining 46 weeks.

About the Global Phase 3 Study

The Global Phase 3 study of NGENLA(r) in 224 treatment-na?ve children with pediatric GHD in over 20 countries was a 12-month randomized, open-label, active-controlled study evaluating the safety and efficacy of weekly NGENLA(r) (somatrogon) injection compared to GENOTROPIN (somatropin) administered once-weekly. Eligible patients were randomized 1:1 into two arms: somatrogon administered at a dose of 0.66 mg/kg body weight once-weekly vs GENOTROPIN(r) (somatropin) administered at a dose of 0.034 mg/kg body weight once-daily.