Growth Spurts: Long-Acting Growth Hormone Revolutionizes Deficiency Treatment

Today, over 200 specialty drugs are on the market, with more than 500 in the works. Just 15 years ago, there were fewer than 30. These drugs, like recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1), are changing how we treat growth hormone deficiency. They’re especially important for kids and adults who need them.

Specialty drugs are now a big part of healthcare spending. They’re expected to hit over $1 trillion a year by 2030. This shows how big an impact they have on treating growth hormone deficiency and other conditions.

Key Takeaways

Specialty drugs, including rhGH and rhIGF-1, have seen a surge in availability and usage for treating childhood short stature and growth hormone deficiency.
The costs of specialty drugs are growing rapidly, with a 10% or more annual increase compared to non-specialty drugs.
rhGH was approved by the FDA in 1985 for treating childhood growth hormone deficiency, a condition affecting an estimated 1 in 3,500 children in the US.
Potential annual costs for rhGH therapy could exceed $10 billion if all eligible children were considered for treatment.
rhIGF-1 has an estimated annual market of $200 million in the US and Western Europe for severe primary IGF-1 deficiency, with a potential $1 billion market for non-severe cases.

What are Specialty Drugs?

Definition and Evolution of Specialty Drugs

Specialty drugs are a special type of medicine that have changed how we treat complex and rare diseases. They need special care, like careful handling and detailed patient education. These drugs are often biologicals, made from genes or proteins, for conditions that affect a few people.

Now, the world of specialty drugs has changed a lot. There are over 200 specialty drugs out there, with more than 500 in the works. This growth has led insurers to add a ‘specialty’ tier, making these expensive drugs cost more for patients.

Today, what makes a drug a specialty drug is often its high price. The Centers for Medicare and Medicaid Services say a specialty drug is any medicine over $600 for a month’s supply. This change shows how big of a deal these specialty drugs are, now a big part of drug spending and biologicals.

“The number of specialty pharmaceuticals has grown exponentially, with over 200 currently available and more than 500 in clinical development, a dramatic increase from 15 years ago.”

Specialty Drugs and Short Stature

Childhood growth is a complex process, influenced by many factors. These include the growth hormone (GH)-insulin-like growth factor-1 (IGF-1) axis. Short stature in children can come from many causes. These include GH deficiency, GH insensitivity, endocrine disorders, systemic diseases, nutritional deficiencies, genetic defects, and family history. The term idiopathic short stature (ISS) is used for short children with no known cause.

To help children with short stature, two specialty drugs are used: recombinant human GH (rhGH) and recombinant human IGF-1 (rhIGF-1). Another drug, leuprolide, can delay puberty in short children. This allows for more growth before puberty starts.

Recombinant human growth hormone (rhGH) is a specialty drug used to treat growth hormone deficiency and idiopathic short stature in children.
Recombinant human insulin-like growth factor-1 (rhIGF-1) is another specialty drug that may be used to treat certain forms of short stature, particularly in cases of GH insensitivity or resistance.
Leuprolide, a gonadotropin-releasing hormone agonist, is a specialty drug that could delay puberty in short children, enabling prolonged growth.

These specialty drugs have changed how we treat childhood short stature. They offer new and more effective ways to help children with growth hormone deficiency and idiopathic short stature.

“The advent of specialty drugs for the treatment of short stature has been a game-changer, offering new hope and improved outcomes for children facing these challenges.”

Recombinant Human Growth Hormone (rhGH)

FDA Approvals and Treatment Impacts

Since 1985, the recombinant human growth hormone (rhGH) has changed how we treat growth hormone deficiency (GHD). Before, the only option was GH from human pituitary glands. This was scarce and risky due to the chance of Creutzfeldt-Jakob disease.

Over 25 years, the FDA has approved rhGH for many conditions that cause short stature. These include chronic kidney problems, Turner syndrome, and being born too small. This has given patients a safe and endless supply of GH, solving the old treatment’s problems.

Studies now show that long-acting GH (LAGH) works as well as daily GH (DGH). LAGH types like Sogroya® and NGENLA® are just as good. PEG-LAGH stands out for its better results, especially with lower doses.

Treatment	Efficacy	Safety
PEG-LAGH	Significantly better height velocity and height standard deviation score compared to other LAGH formulations and DGH	Reduced risk of adverse events compared to other LAGH formulations, comparable with DGH
Other LAGH (somatrogon, somapacitan, lonapegsomatropin)	Comparable efficacy to DGH	Comparable safety profile to DGH
Daily Growth Hormone (DGH)	Established efficacy	Established safety profile

Long-acting GH has made it easier for patients to stick with their treatment. This is because they don’t need to inject themselves every day. These new forms of recombinant human growth hormone have made treating growth hormone deficiency better and easier for patients.

“The development of a sustained-release recombinant human growth hormone formulation has shown promising results, with the potential to revolutionize the treatment of growth hormone deficiency.”

Growth hormone deficiency, long-acting GH

Growth hormone deficiency means the body can’t make enough growth hormone. This hormone is key for normal growth and development. Traditional treatments used recombinant human growth hormone (rhGH). But, long-acting GH has changed how we treat this condition.

Long-acting GH, like somatropin, is a new option. It’s easier to use than daily injections of traditional rhGH. This could lead to better growth for patients with growth hormone deficiency.

The FDA approved NGENLA (somatrogon-ghla) for kids over three with growth issues. It’s a once-a-week human growth hormone. This was after a study showed it worked as well as daily somatropin.

Statistic	Value
Growth hormone deficiency affects	1 in approximately 4,000 to 10,000 children
NGENLA is approved for pediatric GHD treatment in	more than 40 markets around the world, including Canada, Australia, Japan, and EU Member States
Side effects of NGENLA treatment in children with GHD included	injection site reactions, pain, swelling, rash, itching, bleeding, common cold, headache, fever, anemia, cough, vomiting, decreased thyroid hormone levels, stomach pain, rash, and throat pain

Long-acting GH like NGENLA could greatly help children with growth hormone deficiency. It offers a simpler and possibly more effective way to treat this condition than daily injections of traditional rhGH.

Recombinant Human Insulin-like Growth Factor-1 (rhIGF-1)

FDA Approval and Potential Uses

Recombinant human insulin-like growth factor-1 (rhIGF-1) is a new hope for treating growth disorders and metabolic issues. The FDA approved it in 2005 for severe IGF-1 deficiency or growth hormone gene deletion with development of neutralizing antibodies to growth hormone. This treatment could help with many health problems.

Research shows rhIGF-1 can stop motor neuron cell death and help kids with growth hormone insensitivity. It also makes patients taller. Long-term use has been successful in improving growth in kids with growth hormone insensitivity syndrome.

rhIGF-1 might also help with diabetes, insulin resistance, brain repair, and neuroprotection. It can improve how the body uses glucose and insulin, making it a possible solution for insulin-related disorders.

Outcome Measure	Pre-treatment	Post-treatment
Fasting Plasma Glucose (mg/dL)	203 ± 12	134 ± 14
Day-long Plasma Glucose (mg/dL)	234 ± 11	153 ± 10
Basal Endogenous Glucose Production (mg/kg lean body mass·min)	3.2 ± 0.2	2.7 ± 0.2
Fasting Plasma Insulin (μU/mL)	13 ± 5	5 ± 1
Whole Body Insulin-stimulated Glucose Disposal (mg/kg lean body mass·min)	5.6 ± 0.8	7.1 ± 0.8
Hemoglobin A1c (%)	11.7 ± 1.1	N/A

Studies show rhIGF-1 is promising for treating growth and metabolic disorders. As research goes on, we expect to see more ways to help patients.

“Long-term treatment with recombinant insulin-like growth factor-I (IGF-I) in children with severe IGF-I deficiency due to growth hormone insensitivity showed positive results with a range of 128-134.”

Pros and Cons of Specialty Drugs for Short Stature

Specialty drugs like recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1) therapy have both good and bad sides. It’s important to know these points to make the right choice for treatment.

RhGH therapy is great because it helps kids grow taller. It works well for kids with growth hormone deficiency and other short stature conditions. Kids can grow 1 to 2 inches taller in the first 6 months, and they might grow even more over time. This therapy also makes kids leaner and less fat, which is good for their health.

But, these drugs are very expensive. The cost for a year of rhGH or rhIGF-1 therapy can be $20,000 to $30,000 per child. This price can stop some families from getting the treatment they need.

RhGH therapy is usually safe, but it can cause some side effects. These include allergic reactions, joint pain, headaches, and higher blood sugar levels. We’re still learning about the long-term effects on health and how it affects mental well-being and life expectancy.

Choosing to use specialty drugs for short stature means looking at the good and bad sides. It’s important to think about the benefits and costs with a doctor’s help. This way, you can make the best choice for you or your child.

Longitudinal studies on chronic diseases help us understand how these drugs work over time. They tell us about their safety, how well they work, and if they’re worth the cost.

“The use of specialty drugs for short stature management is a complex decision that requires carefully weighing the pros and cons to ensure the best possible outcomes for the patient.”

Height Attainment with rhGH Therapy

The use of recombinant human growth hormone (rhGH) therapy has been found to increase final height in kids with growth hormone deficiency (GHD) and other conditions. For kids with classical GHD, rhGH therapy makes growth speed up and helps them reach normal adult height if started early. They gain about 12 inches on average.

In other conditions, rhGH therapy leads to smaller height gains. For example, kids with chronic renal insufficiency gain 1-4 inches, and those with Prader-Willi syndrome gain 7-9 inches. Most kids with certain conditions grow to be within the normal height range. However, this is not always the case for kids with Turner syndrome or SHOX deficiency.

Studies show that the mean gain in final height SDS after GH treatment in GHD is 1.5-2.0 for average cases and 3.5 for extreme cases.
In GHD kids who started puberty naturally, the final height SDS was -2.8. For those who had puberty induced, the mean final height was -1.6.
For kids with idiopathic short stature, a GH dosage of 27 IU (9 mg)/m2·wk led to a mean final height gain of about 7 cm. Lower dosages were less effective.

Overall, rhGH therapy has shown significant improvements in height attainment for kids with various growth issues. Many kids with these conditions now reach adult heights within the normal range.

“The difference between final height and predicted adult height was significantly greater in GH-treated children (4.0 cm) compared to controls (0.8 cm).”

Other Physical Effects of rhGH Therapy

Recombinant human growth hormone (rhGH) therapy does more than help kids grow taller. It also has metabolic effects on body composition, bone density, and heart health.

Research shows that rhGH can make the body leaner by increasing muscle and reducing fat. It also helps make bones stronger by increasing bone mineral density. This is important for keeping bones healthy and strong.

But, the safety of using rhGH for a long time is still being studied. Doctors need to watch patients closely to make sure it’s safe and effective for them.

Outcome	Findings
Bone Mineral Density	Studies over a year showed more bone mineral density in the spine and hip. The spine and hip bones got stronger by a certain amount. Shorter studies didn’t show much change in bone density. Studies looking ahead showed more bone density in the spine and hip. Younger patients and longer treatment got bigger benefits. Men saw bigger gains in bone density than women.

It’s important to keep studying the safety and metabolic effects of rhGH therapy. Doctors need to check on patients often to make sure they’re getting the best care.

Challenges in Short Stature Treatment

Specialty drugs like recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1) have changed how we treat short stature in kids. But, there are still big hurdles to overcome for getting these treatments to those who need them.

These medicines are very expensive, costing between $20,000 to $30,000 a year for each child. If all eligible kids got treatment, it could cost over $10 billion a year in the U.S. alone. There’s also ongoing research to make sure these drugs are safe and work well for more than just the approved uses.

It’s important to make sure these treatments are used right, easily available, and give the best value. Solutions like genetic testing and using AI in healthcare could help solve these problems. They could also improve the lives of kids with short stature.

Challenges	Impact
High Cost of Specialty Drugs	Annual prices ranging from $20,000 to $30,000 per child, potentially exceeding $10 billion in annual costs in the U.S.
Long-term Safety and Efficacy	Ongoing research and monitoring required for use beyond approved indications
Ensuring Appropriate Use and Access	Critical priority for policymakers, medical community, and society

The progress in treating short stature is ongoing. But, we must tackle these challenges to make sure every child gets the care they deserve. This way, they can reach their full potential.

Case Study: Refractory Juvenile Idiopathic Arthritis

Juvenile idiopathic arthritis (JIA) is a tough autoimmune condition. It can be hard to treat, especially when it doesn’t respond to usual treatments. This story is about a 16-year-old girl who had JIA that didn’t get better with many treatments. She got a surprising diagnosis and needed help from many doctors.

The girl had high inflammation markers like an ESR of 129 mm/1st h and a CRP of 226 mg/l. She tried many treatments like nonsteroidal anti-inflammatory drugs, glucocorticoids, methotrexate, and biologic agents. But, her symptoms got worse, causing her to grow less and develop osteoporosis due to too much inflammation and steroid use.

Then, genetic tests found a problem with the short-stature homeobox gene. This meant she had Leri Weill syndrome, a rare condition causing short stature and Madelung deformity. This finding showed how important it is to look for other possible causes when treatments don’t work.

This case shows how important it is to work together to treat complex diseases like JIA. It also shows the value of sharing stories of hard cases. This helps doctors understand and treat similar cases better, especially when they involve conditions like Leri Weill syndrome.

Navigating Refractory Disease

The girl tried many treatments, including tofacitinib, a JAK inhibitor for tough JIA cases. But, her condition was still hard to manage. She faced growth issues, osteoporosis, and needed several surgeries.

“This case highlights the need for reporting and sharing experiences with refractory, complicated cases in order to build knowledge and provide guidance for physicians treating common rheumatological diseases like JIA, especially when additional diagnoses are involved.”

When she moved from pediatric to adult care, it was tough. She hadn’t had a break from her illness. So, she needed a special plan to keep her care consistent and manage her disease well.

This story reminds us of the challenges in treating JIA that doesn’t respond to usual treatments. It’s important to look for other possible causes. By sharing these stories, we can help improve care for patients with this serious condition.

Conclusion

Specialty drugs like recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1) have changed how we treat short stature in kids. These drugs offer new ways to help kids with growth hormone deficiency and growth hormone insensitivity. They could lead to better growth and quality of life. But, these drugs are very expensive, costing between $20,000 to $30,000 a year for each child.

This high cost makes it hard to make sure all kids can get these drugs. The long-term effects of these treatments are still being studied. As more kids use these drugs, we need to work together to make sure they are used wisely. This ensures they help patients the most and are worth the cost.

New long-acting GH therapies, like TransCon GH and Somapacitan, aim to make treatment easier and overcome daily injection challenges. As these new treatments grow, we must check how well they work, how safe they are, and how they affect patients. This will help make sure they really change the lives of kids and adults with growth issues.

FAQ

What are specialty drugs?

Specialty drugs are special medicines that need careful handling and expert care. They often come with a higher price tag. Many are biologicals, made through genetic or protein engineering.

How have specialty drugs evolved?

At first, specialty drugs were for rare, complex conditions. Now, over 200 are available, with more than 500 in the works. This growth has made them a big concern for healthcare costs, as they’re rising fast.

What specialty drugs are used to treat childhood short stature?

Two drugs help treat short stature in kids: recombinant human growth hormone (rhGH) and recombinant human insulin-like growth factor-1 (rhIGF-1).

When was recombinant human growth hormone (rhGH) first approved?

The FDA approved rhGH in 1985 for kids with growth hormone deficiency. This condition affects about 1 in 3,500 kids in the US. Before, GH came from human pituitary glands, but it was hard to get and risky.

How has the development of long-acting GH formulations impacted the treatment of growth hormone deficiency?

Long-acting GH, like somatropin, changed how we treat growth hormone deficiency. It’s easier and might work better, helping kids grow better and stick to their treatment.

When was recombinant human insulin-like growth factor-1 (rhIGF-1) approved?

Human IGF-1 was cloned in 1983 and made in labs in 1986. Studies showed it helped kids with certain growth issues. The FDA approved it in 2005 for kids with severe growth problems.

What are the potential benefits of rhGH and rhIGF-1 therapy for short stature?

rhGH and rhIGF-1 help kids with short stature grow taller. They also improve metabolism, bone density, and heart health.

What are the challenges in the use of specialty drugs for short stature?

These drugs are expensive, costing ,000 to ,000 a year for each child. This makes them hard to get for many families. There’s also ongoing research on their long-term safety and effectiveness.

What is the case study about?

The case study is about a 16-year-old girl with arthritis that didn’t respond to many treatments. Genetic tests showed she had Leri Weill syndrome. It shows the importance of sharing complex case stories to help doctors treat similar conditions.