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In recent years, with the increasing application of molecular biology technology in the medical field, the diagnosis and treatment of diseases in the field of pediatric endocrinology, especially rare diseases, has developed rapidly, but there are still challenges
in the diagnosis and treatment of some diseases such as childhood short stature.
Professor Li Yanhong, deputy chief physician of the Department of Pediatrics of the First Affiliated Hospital of Sun Yat-sen University, said
in a recent interview with the pharmaceutical melting circle.
Since 1999, Professor Li Yanhong has carried out clinical and research work in the field of pediatric endocrinology, she revealed to the pharmacfinance circle that the field of pediatric endocrinology covers the diagnosis and treatment of a variety of endocrine diseases and genetic metabolism (rare diseases), but at present, the most common in pediatric endocrinology and child health clinics is still precocious puberty and short stature
.
Among them, for short stature, although the diagnosis of the disease has the help of molecular biology, which greatly improves the level of precision treatment, there are still problems
in the diagnosis and treatment of growth hormone deficiency in children.
Growth hormone deficiency in children urgently requires early diagnosis and early treatment
Growth hormone deficiency in children urgently requires early diagnosis and early treatmentProfessor Li Yanhong introduced that statistically speaking, the category of short stature means that the height of children is less than 2 standard deviations of the average height of healthy children of the same race, age and sex, or lower than the third percentile of the growth curve
.
Of the estimated 250 million children in the country, about 7 million suffer from short stature, but less than 5%
are likely to be diagnosed and treated promptly.
Growth hormone secreted by the anterior pituitary gland (hGH) plays a very important role in the influence of height, and is also one of the most important endocrine hormones in the process of individual growth and development, growth hormone deficiency (GHD), also known as pituitary dwarfism, is an important cause
in childhood short stature 。 Professor Li Yanhong explained: "Unlike other short stature, growth hormone deficiency is caused by organic lesions or functional abnormalities of the hypothalamic-pituitary axis caused by growth hormone deficiency, in addition to low height growth rate and short stature, there will be other problems caused by growth hormone deficiency, including long-term and adult metabolism, bone health and development and other problems, which are diseases that require long-term growth hormone replacement therapy, and the improvement effect of short stature after treatment is the best, but early diagnosis and early treatment are required.
"
。 ”
In 2021, the National Health Commission issued the Action Enhancement Plan for Healthy Children (2021-2025), proposing to control the stunting rate of children under 5 years old below 5% by 2025
.
However, due to the imbalance in the level of medical care in different regions, children with growth hormone deficiency have different opportunities to be diagnosed and receive early diagnosis and treatment
.
Professor Li said: "In Guangdong Province, there are relatively many institutions or specialized hospitals and professionals who have set up pediatric endocrinology specialties, including GHD, and short children can be diagnosed and treated early, but there is a lack of pediatric endocrinology specialists or related personnel in some grassroots areas, and general doctors do not understand children's short stature, and popular science education may not be in place, and there are certain deficiencies in the diagnosis and treatment of growth hormone deficiency, as well as the monitoring of the efficacy of growth hormone treatment, including the course of treatment, side effects, and long-term management
。 "Obviously, there is still a big gap
between the diagnosis and treatment of growth hormone deficiency and the relevant national action goals to pay attention to children's health.
With the successful development of recombinant growth hormone, the production capacity of growth hormone has increased significantly, and more and more patients are able to access treatment
.
However, in recent years, controversies over growth hormone have begun to emerge, and even so-called growth hormone "abuse" arguments have
emerged.
In this regard, Professor Li Yanhong believes that it is necessary to look at it from two aspects:
First of all, with the increase of growth hormone sources, its indications have gradually expanded, in addition to growth hormone deficiency, but also gradually extended to other diseases, including Turner syndrome, Noonan syndrome, PWS, small-for-gestational-age infants, idiopathic short stature, etc
.
"For GHD and PWS, Turner syndrome, etc.
, GH treatment has other benefits in addition to improving height, which is already relatively clear, so these children should be
treated.
" But other short stature, especially idiopathic short stature (ISS) is controversial, these children short stature causes are complex, growth hormone is not deficient, but in recent years more and more studies have shown that after growth hormone treatment, can obtain adult height improvement, and there are no long-term serious side effects, ISS is also included in the indication range of growth hormone
.
Professor Li said
.
However, in recent years, there have also been cases where parents have high expectations for their children's height and actively request GH treatment, although growth hormone is safe and effective, but does not mean that growth hormone can be abused, short children should be carefully evaluated and standardized in a regular pediatric endocrinologist to evaluate and standardize diagnosis and treatment, the treatment should be treated, should not be treated is not
forced.
Some people hear that there is "abuse" of growth hormone, but those who should be treated are not willing to treat, so that the opportunity for treatment is lost, which is a pity
.
Professor Li sighed
.
Long-acting preparations of growth hormone are imperative
Long-acting preparations of growth hormone are imperative"When it comes to growth hormone therapy, we have to mention the issue of long-acting," Professor Li Yanhong mentioned
.
The earliest growth hormone was necrogenic, but was banned in 1985 due to viral contamination
.
By the end of the 80s of last century, recombinant growth hormone entered people's attention and began subsequent industrial production, and safe and effective growth hormone preparations were applied to patients
.
In the mid-to-late 90s, imported growth hormone was very expensive
.
"At that time, there were very few children who could use growth hormone, so sometimes we were very sad that some children were diagnosed with growth hormone deficiency, but they really did not have the financial means to treat
it.
" Professor Li said
.
With the development of domestic growth hormone, more and more children with GHD can now be treated, and a major feature of growth hormone therapy is that it requires long-term treatment, and there is no oral preparation for injection, and daily injection
is required in the past.
Professor Li Yanhong said to the pharmaceutical circle: "If it is a growth hormone deficiency, the patient must be diagnosed from the age of four or five to start the injection until puberty, the period of more than 10 years, the pain caused by it can not be ignored, the past daily injection and long-term treatment affect the patient's
treatment compliance.
" ”
Foreign studies have shown that 66% of patients miss needles once a week
, especially for people who need to start treatment at a young age (including GHD, SGA, ISS, etc.
), it is difficult to adhere to
.
And the more needles missed, the worse the growth rate
.
Even though in recent years there is a desire to improve the injection experience and increase compliance through the development of pens that facilitate injection, improved formulas, and storage methods, there are still a considerable number of children whose compliance is compromised
due to the need for daily injections.
"So overall, long-acting growth hormone is imperative
.
" Professor Li Yanhong said
to the medicine melting circle.
Foreign countries have long begun the research and development of long-acting growth hormone, and in the 21st century, some bottlenecks in the development of long-acting preparations have been broken, especially the research and development of polyethylene glycol (PEG) long-acting long-acting growth hormone has been successful, making long-acting growth hormone products successfully commercialized
.
The advent of long-acting preparations has reduced the number of injections per year from more than 350 injections per year to only 52 injections per year, reducing to about one-sixth of the previous level, and patient compliance has been greatly improved
.
Longpei auxin "beats" daily preparations for the first time
Longpei auxin "beats" daily preparations for the first timeIn November 2022, Veson Pharmaceutical, an innovative biopharmaceutical company focusing on endocrine-related therapeutics, announced its long-acting growth hormone (weekly preparation), lonapegsomatropin,
TransCon hGH) Phase 3 pivotal clinical trial in Chinese children with growth hormone deficiency (GHD
).
Longpei auxin is the only growth hormone prodrug developed using TransCon temporary ligation technology in the world to release unmodified growth hormone
in the human body through weekly administration.
The results showed that the annualized growth rate (AHV) of 52 weeks of treatment with Longpei auxin or growth hormone was 10.
66 cm/year and 9.
75 cm/year, respectively (the difference between groups was 0.
91, 95%
Confidence interval: 0.
37 ~ 1.
45, p=0.
0010), the primary endpoint of the study was achieved, that is, longpei auxin was not inferior to daily preparation of growth hormone; And achieve efficiency
.
66 cm/year and 9.
75 cm/year, respectively
This trial is a randomized, open-label, active controlled clinical trial for the treatment of growth hormone deficiency in children in China, and the analysis results of the primary endpoint also confirmed the superiority of growth hormone daily preparation
based on preset statistical tests.
Multiple preset sensitivity analyses confirmed the robustness
of the results.
Safety results showed that Longpei auxin was well tolerated and the safety profile was comparable
to that of daily preparations of growth hormone.
Neutralizing anti-drug antibody production
has not been observed in global clinical trials.
That is, for the first time, Longpei auxin "defeated" the daily preparation of growth hormone
.
Longpei auxin is the only product currently validated by a well-designed randomized controlled trial comparing long-acting versus a daily HGH formulation compared to previous products that have used historical controls or concluded from trials from different investigators
.
Professor Li Yanhong, as an important participant in the phase 3 key clinical trial in China, has a very intuitive feeling about Longpei auxin, she said: "The advent of polyethylene glycol (PEG)ated growth hormone can be said to be a milestone! Longpei auxin is also a leader in PEGylated growth hormone preparations and all other long-acting growth hormone preparations
.
”
After entering the human body, Longpei auxin releases the same molecular form as the natural growth hormone of the human body, while other modified growth hormone preparations entering the human body cannot fully function
in the structure of growth hormone closest to the prototype.
Therefore, after understanding and understanding the mechanism of action of TransCon technology, Professor Li expressed his affirmation of this innovative technology: "I think TransCon technology is indeed very great, because the technology of "temporary connection" can be realized, so that the modified GH can release growth hormone with the closest structure to natural GH after dissociation in the human body, and maximize the physiological efficacy
of growth hormone in the human body.
" It can be said that the development of Longpei auxin using TransCon technology should be the most advantageous long-acting preparation of growth hormone at present
.
For such a technology platform, not only growth hormone, if it is applied to other hormones
or molecules for such a temporary combination, it is also very potential.
”
In response to this randomized controlled trial, Professor Li Yanhong mentioned that daily injection of growth hormone has become a conventional drug for GHD treatment after many years of application, and the efficacy is undoubted
.
For example, a 6-year-old child with a growth rate of less than 4 cm/year before treatment can achieve a growth rate
of 10 cm/year after daily injection.
When a new long-acting formulation enters the research in this field, it is difficult to judge whether the long-acting preparation is superior to the daily preparation
if it is simply concluded that the new formulation can achieve a growth rate of 10cm/year.
Longpei auxin is a randomized controlled trial, compared with the daily preparation, the annual growth rate difference after treatment is about 1~2cm, "This is quite remarkable, with the comparison is more convincing
.
" Previous studies of new drugs have only shown how much benefit they bring to patients, and have not been compared head-to-head with daily preparations, so it cannot be absolutely said that they are better
than daily preparations.
This time the clinical trial set up a control group, the data is reliable and more scientific
.
Randomized controlled trials may also be needed in future long-acting formulations
.
Professor Li Yanhong said
.
.
Randomized controlled trials may also be needed in future long-acting formulations
Overall, Longpei auxin has the potential to be the best in long-acting growth hormone formulations due to its structural advantages and characteristics of drug metabolism
class products
.
Professor Li Yanhong added: "Longpei auxin is good news for the medical community and patients, because the first consideration of drug development is efficacy, safety, and secondly convenience of use
.
I hope that with the release of China's phase 3 clinical trial data, the product can be promoted and marketed in China as soon as possible, and I hope that patients can have more opportunities to obtain good therapies, reduce the pain of child injection, and make children happier! ”
This article is only for providing scientific information to healthcare professionals and does not represent the position of the platform
This article is only for providing scientific information to healthcare professionals and does not represent the position of the platformReference:
Reference:NMPA/CDE；
NMPA/CDE；
YaoRong Cloud Data, www.
pharnexcloud.
com;
pharnexcloud.
com;
FDA/EMA/PMDA；
FDA/EMA/PMDA；Public disclosure by relevant companies;
Public disclosure by relevant companies;；
；；
；
https://mp.
weixin.
qq.
com/s/mY8Wm5krZpcIQ3XEtOrCag； And so on
.
weixin.
qq.
com/s/mY8Wm5krZpcIQ3XEtOrCag； And so on
.
