Ngenla

Pediatric Growth Hormone Deficiency

Ngenla is used to treat children 3 years of age and older who have growth failure caused by a lack of adequate growth hormone production in the body. This condition, known as growth hormone deficiency (GHD), occurs when the pituitary gland does not release enough growth hormone to support normal height and development.

Purpose of Treatment

The primary goal of using Ngenla is to provide a supplemental source of growth hormone for children whose bodies cannot make enough on their own. By replacing this hormone, the medication helps to:

• Increase the rate of linear growth (height).
• Support physical development in children with confirmed growth failure.

Ngenla is a long-acting form of growth hormone, which distinguishes it from some other treatments that may require more frequent administration. It is intended for long-term use under the supervision of a healthcare professional specializing in pediatric growth disorders.

Mechanism of Action

Ngenla belongs to a class of medications known as growth hormones. It is a laboratory-made version of the growth hormone naturally produced by the pituitary gland. In children with growth hormone deficiency, the body does not produce enough of this hormone to support normal physical development.

Ngenla works by mimicking the effects of natural growth hormone in the body through the following process:

• Receptor Binding: Once injected, the medication binds to specific growth hormone receptors on the surface of cells.
• Stimulating Growth Factors: This binding triggers the release of insulin-like growth factor-1 (IGF-1), a protein that is essential for childhood growth.
• Promoting Development: Growth hormone and IGF-1 work together to signal the bones and other tissues to grow, helping the child increase their height and reach developmental milestones.

Ngenla is specifically engineered to be long-acting. It remains active in the bloodstream for an extended period, which allows it to provide a steady supply of the hormone with one injection per week, rather than the daily injections required by some other growth hormone treatments.

Common Side Effects

The most frequently reported side effects of Ngenla include:

• Injection site reactions, such as pain, redness, swelling, or itching where the shot was given
• Headache
• Common cold symptoms, such as a runny nose or sore throat
• Fever
• Anemia (low red blood cell count)
• Joint or back pain

Serious Side Effects

While less common, some side effects can be serious. Contact a healthcare provider immediately if any of the following occur:

• Severe allergic reactions: Symptoms may include rash, hives, swelling of the face, lips, or tongue, and difficulty breathing.
• Increased pressure in the brain: This may cause vision changes, severe headaches, nausea, or vomiting.
• High blood sugar: Signs include increased thirst, frequent urination, or fruity-smelling breath.
• Bone and joint issues: A new or worsening limp, or pain in the hip or knee.
• Adrenal gland problems: Symptoms can include extreme tiredness, dizziness, or weight loss.
• Pancreatitis: Severe pain in the upper stomach area that may spread to the back.
• Progression of scoliosis: Worsening of a pre-existing curve in the spine.

Contraindications

Ngenla is contraindicated in patients with the following conditions:

• Active malignancy (cancer), as growth hormone may promote the growth of certain tumors.
• Acute critical illness resulting from complications of major surgery, multiple accidental trauma, or acute respiratory failure.
• Closed epiphyses, meaning the child's growth plates have already fused and they can no longer grow in height.
• Active proliferative or severe non-proliferative diabetic retinopathy.
• Known hypersensitivity to somatrogon-ghla or any of the inactive ingredients in the medication.

Severe Hypersensitivity

Serious systemic allergic reactions, including anaphylaxis and angioedema, have been reported with growth hormone products. If a serious allergic reaction occurs, the medication should be stopped immediately and medical care sought.

Increased Intracranial Pressure

Intracranial hypertension (increased pressure within the skull) can occur during treatment. Symptoms may include persistent headache, nausea, vomiting, or visual changes. Healthcare providers typically monitor for this by performing eye examinations before and during therapy.

Metabolic and Endocrine Effects

Treatment with Ngenla may reduce insulin sensitivity, which can lead to glucose intolerance or the development of type 2 diabetes. Regular monitoring of blood glucose levels is recommended, especially for patients with existing risk factors. Additionally, growth hormone therapy may affect thyroid function or adrenal hormone levels, requiring periodic blood tests and potential adjustments to other medications.

Skeletal and Joint Issues

Rapid growth during therapy may lead to the progression of preexisting scoliosis. Patients may also experience a slipped capital femoral epiphysis, a condition where the ball of the hip joint slips. Healthcare providers should be notified if a patient develops a limp or experiences hip or knee pain.

Pancreatitis

Cases of pancreatitis (inflammation of the pancreas) have been reported in patients receiving growth hormone. This condition should be considered if a patient develops severe and persistent abdominal pain.

Lipoatrophy

To avoid the loss of fat tissue at the injection site (lipoatrophy), it is important to rotate the site of injection with each dose.

Pregnancy

It is currently unknown whether Ngenla can cause harm to an unborn baby. Clinical trials have not specifically studied the use of this medication in pregnant individuals. If a patient becomes pregnant or plans to become pregnant while using Ngenla, they should consult their healthcare provider to discuss the potential risks and benefits of continuing treatment.

Breastfeeding

It is not known if Ngenla passes into breast milk or if it could affect a nursing child. Because the effects on a breastfeeding infant are uncertain, it is important for patients to talk with their doctor about the risks and benefits of breastfeeding while receiving this medication.

Available Forms

Ngenla is supplied as a sterile solution for injection. It is available in the following delivery format:

• Prefilled Pen: A single-patient-use, disposable injection device containing a clear to slightly yellow liquid solution.

The medication is designed for subcutaneous injection once per week. The prefilled pen is a multi-dose device intended for use by a single patient. Once the pen is empty or has reached the end of its use period after being opened, it must be disposed of according to safety guidelines.

FDA Approval Status

The U.S. Food and Drug Administration (FDA) approved Ngenla on June 27, 2023. It is classified as a human growth hormone analog. The medication is specifically indicated for the treatment of pediatric patients 3 years of age and older who have growth failure due to an inadequate secretion of endogenous growth hormone.

Regulatory History

Ngenla was developed as a long-acting alternative to daily growth hormone injections. Its approval was based on clinical evidence demonstrating its safety and efficacy in promoting growth in children with growth hormone deficiency. The FDA approval provides a once-weekly subcutaneous injection option for this patient population.

Safety and Monitoring

At the time of approval, the FDA did not require a boxed warning or a Risk Evaluation and Mitigation Strategy (REMS) for Ngenla. As with other growth hormone products, regulatory authorities monitor the drug for potential side effects such as injection site reactions, headaches, and joint pain. Healthcare providers are expected to monitor patients regularly for growth response and potential adverse events during treatment.

Factors Affecting Cost

The actual price paid for Ngenla depends on several variables. These include the type of insurance plan, the specific pharmacy dispensing the medication, and the dosage required based on the patient's weight.

Insurance and Prior Authorization

Many insurance companies require prior authorization for Ngenla. This means the healthcare provider must submit documentation explaining why the medication is medically necessary before the insurance company will agree to cover the cost. Out-of-pocket costs like copays and deductibles will vary by plan.

Financial Assistance

Financial assistance may be available for eligible patients to help manage the cost of Ngenla:

• Copay Cards: The manufacturer may offer savings cards for patients with commercial insurance to reduce out-of-pocket expenses.
• Patient Assistance Programs: For those without insurance or whose insurance does not cover the drug, programs may provide the medication at a reduced cost or for free based on financial need.

Because Ngenla is a specialty medication, it is typically not available at all retail pharmacies. Patients may need to use a specialty pharmacy designated by their insurance provider.