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Pfizer/OPKO long-acting human growth hormone BLA
Pfizer/OPKO long-acting human growth hormone BLA. On January 4, 2020, Pfizer and OPKO Health announced that the FDA has accepted somatrogon’s biological product licensing application (BLA). Somatrogon is a long-acting human growth hormone once a week for the treatment of growth hormone deficiency (GHD) in children.
Somatrogon (hGH-CTP) is glycosylated and consists of the amino acid sequence of human growth hormone and the 28 amino acids of the C-terminal peptide of human chorionic gonadotropin β subunit (CTP). Specifically, one CTP copy of human chorionic gonadotropin β subunit was added to the N-terminal of growth hormone, and two CTP copies were added to the C-terminal. Compared with current growth hormone replacement therapy, this design aims to reduce the frequency of injections from once a day to once a week.
OPKO Health’s Carboxyl Terminal Peptide (CTP) technology is used to extend the half-life of protein drugs. CTP uses recombinant technology to fuse a naturally-occurring 28 amino acid sequence with protein to slow down protein excretion, thereby extending the half-life of protein. In addition to GHD, OPKO currently also uses CTP technology in factor VIIa products to treat hemophilia A and B.
Pfizer and OPKO reached a cooperation agreement in 2014 on the development and commercialization of somatrogon for GHD. According to the agreement, OPKO is responsible for the implementation of clinical projects, while Pfizer is responsible for product registration and commercialization.
This application is based on positive data from a phase III clinical trial. This randomized, open-label, positive-controlled study was carried out in more than 20 countries/regions, and a total of 224 children with GHD who had not previously received treatment were recruited and treated. These patients were randomly divided into two groups at a ratio of 1:1: taking somatrogon at a dose of 0.66 mg/kg body weight once a week, and taking GENOTROPIN® (somatropin) at a dose of 0.034 mg/kg body weight once a day.
The main endpoint of the study is the height velocity at 12 months. Secondary endpoints include changes in height standard deviation at 6 months and 12 months, safety and pharmacodynamic indicators. Children who complete this study have the opportunity to be enrolled in an open-label, multi-center, long-term expansion study, and can continue to receive or switch to somatrogon. Approximately 95% of patients entered the open-label extension study and received somatrogon treatment.
By measuring the growth rate of height at 12 months, the study reached the primary endpoint of non-inferiority compared with somatropin injected once a day. Somatropin has a history of decades, but daily injections may cause patients to lack compliance.
The top-line results of the study showed that the least squares mean of the somatrogon group was significantly higher than that of the somatropin group, which were 10.12 and 9.78 cm/year, respectively; the therapeutic difference in the rate of height growth was 0.33 (95% CI 0.39-1.05). In addition, the researchers pointed out that compared with somatropin, treatment with somatrogon was associated with higher changes in height standard deviation scores at 6 and 12 months.
In terms of a key secondary endpoint, the somatrogon group had a higher rate of height growth at 6 months compared with the somatropin group. In addition, the somatrogon treatment in the study was generally well tolerated, comparable to somatropin, especially in terms of the type, number and severity of adverse events.
(source:internet, reference only)