Japan’s MHLW Approves Pfizer and OPKO’s NGENLA® (Somatrogon), a New Long-Acting Treatment for Pediatric Growth Hormone DeficiencyNGENLA® offers a new once-weekly treatment option for children living with growth hormone deficiency

2022-01-20

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Pfizer Japan Inc. and OPKO Health, Inc. (NASDAQ: OPK) announced today that the next generation long-acting growth hormone injection, NGENLA® (somatrogon) Inj. 24 mg Pens and 60 mg Pens, has been approved by the Ministry of Health, Labour and Welfare (MHLW) in Japan. NGENLA® 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® 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® with GENOTROPIN® (somatropin), a recombinant human growth hormone for injection administered once-daily. In both studies, NGENLA® showed comparable efficacy to GENOTROPIN in the primary endpoint of annual height velocity at 12 months. NGENLA® 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®, 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 the Japan Phase 3 Study The Phase 3 study of NGENLA® in 44 treatment-naïve 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® 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® 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® 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® in 224 treatment-naïve 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® (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® (somatropin) administered at a dose of 0.034 mg/kg body weight once-daily.

About NGENLA® (somatrogon) injection NGENLA® is a biologic product that is glycosylated and comprises the amino acid sequence of human growth hormone and one copy of the C-terminal peptide (CTP) from the beta chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies of CTP (in tandem) at the C-terminus. The glycosylation and CTP domains account for the half-life of the molecule. NGENLA® was approved in Canada in October 2021 and in Australia in November 2021.

About GENOTROPIN GENOTROPIN (somatropin) is a man-made, prescription treatment option, approved in the United States for children who do not make enough growth hormone on their own, have the genetic condition called Prader-Willi syndrome (PWS), were born smaller than most other babies, have the genetic condition called Turner syndrome (TS) or have idiopathic short stature (ISS). GENOTROPIN is also approved to treat adults with growth hormone deficiency. GENOTROPIN is taken by injection just below the skin and is available in a wide range of devices to fit a range of individual dosing needs. GENOTROPIN is just like the natural growth hormone that our bodies make and has an established safety profile.

About Growth Hormone Deficiency Growth Hormone Deficiency Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland and affects one in approximately 4,000 to 10,000 people1,2. In children, this disease can be caused by genetic mutations or acquired after birth1,3. Because the patient's pituitary gland secretes inadequate levels of somatropin, the hormone that causes growth, his or her height may be affected and puberty may be delayed1,3,4. Children may also experience other problems with physical health and mental well-being3,4.

Pfizer Rare Disease Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders. Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

OPKO Health, Inc. OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development and commercialization expertise and novel and proprietary technologies.

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