Skytrofa (Previously Lonapegsomatropin Ascendis Pharma)

Treatment should be initiated and monitored by physicians who are qualified and experienced in the diagnosis and management of paediatric patients with GHD. The amount and concentration of lonapegsomatropin is always expressed in terms of mg somatropin referring to the content of the somatropin moiety and not including mPEG-linker in order to prevent medication errors when patients switch from daily somatropin therapy.

4 Posology The posology and administration should be individualised for each patient. 24 mg somatropin/kg body weight, given once weekly. The recommended starting dose strengths for such a dose by weight range can be found in Table 1.

24 mg somatropin/kg/week, calculate the total weekly dose (in mg somatropin) and select the appropriate dose strength as follows: • Total weekly dose (mg somatropin) = prescribed dose (mg somatropin/kg) x patient’s body weight (kg) • Round the total weekly dose (mg somatropin) to the closest dose strength while also considering treatment goals and clinical response.

Starting dose for patients switching from daily somatropin medicinal products If changing therapy to once-weekly lonapegsomatropin from daily somatropin, there should be at least 8 hours between the final dose of once-daily somatropin and the first dose of lonapegsomatropin.

In children switching from daily somatropin, physicians may adjust the starting dose taking into consideration the current somatropin dose, individual clinical response, and clinical considerations specific to the patient. 24 mg somatropin/kg body weight (Table 1).

24 mg somatropin/kg body weight, use the previously prescribed weekly dose as the recommended starting dose of lonapegsomatropin (see equation above). Dose titration The dose of lonapegsomatropin should be individually adjusted for each patient based on clinical response, adverse reactions, and/or serum insulin-like growth factor-1 (IGF-1) concentrations outside the targeted range.

Available somatropin dose strengths can be found in section 1. 5 Average IGF-1 standard deviation score (SDS) levels (drawn 4-5 days after dosing) can be used as guidance for dose titration (Table 2). It is necessary to wait a minimum of 2 weeks after initiation of lonapegsomatropin or after any dose change before assessing the resulting IGF-1 SDS levels.

6%). In general, these reactions tended to be transient, and severity was mild to moderate. Tabulated list of adverse reactions Table 3 below shows adverse reactions which occurred during lonapegsomatropin treatment. The adverse reactions are ranked under headings of MedDRA system organ class and frequency using the following terminology: very common (≥1/10), common (≥1/100 to <1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000), and frequency not known (cannot be estimated from the available data).

Table 3 Frequency of adverse reactions in clinical trials System organ class Very common Common Uncommon Immune system disorders Anaphylactic reactionb Endocrine disorders Secondary hypothyroidism Secondary adrenocortical insufficiency Nervous system disorders Headache Musculoskeletal and connective tissue disorders Arthralgia Scoliosis Arthritis Growing pains Reproductive system and breast disorders Gynaecomastia General disorders and administration site conditions Injection site reactionsa a Injection site reactions include hyperaemia, injection site atrophy, injection site pain, injection site urticaria, and localised oedema.

The injection site reactions observed with lonapegsomatropin were generally mild and transient. 4). Description of selected adverse reactions Immunogenicity Patients may develop antibodies to lonapegsomatropin. 3%) and no patients had neutralising antibodies.

No apparent correlation of anti-lonapegsomatropin binding antibodies to adverse events or loss of efficacy was observed. 4). 11 Adverse reactions related to growth hormone pharmacological class In addition to the above-mentioned adverse drug reactions, those presented below have been reported with other growth hormone-containing products.

Frequencies of these adverse events cannot be estimated from the available data (unless otherwise indicated). 4). 4). 4), paraesthesia. • Musculoskeletal and connective tissue disorders: myalgia. • Reproductive system and breast disorders: gynaecomastia (frequency: uncommon).

Traceability In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded. e. 1–56 mg/week) compared to patients receiving placebo, 42% vs.

19%. As there is no information available on the safety of growth hormone substitution therapy in 7 acutely critically ill patients, the benefits of continued lonapegsomatropin treatment in this situation should be weighed against the potential risks involved.

In all patients developing other or similar acute critical illness, the possible benefit of treatment with lonapegsomatropin must be weighed against the potential risk involved. Neoplasm In patients with previous malignant disease, special attention should be given to signs and symptoms of relapse.

Patients with pre-existing tumours or GHD secondary to an intracranial lesion should be examined routinely for progression or recurrence of the underlying disease process. In childhood cancer survivors, an increased risk of a second neoplasm has been reported in patients treated with growth hormone after their first neoplasm.

Intracranial tumours, in particular meningiomas, were the most common form of a second neoplasm reported in patients treated with radiation to the head for their first neoplasm. Hypersensitivity Anaphylactic reactions including angioedema have been reported with the use of lonapegsomatropin.

Inform patients and caregivers that such reactions can occur, particularly after first dose, and that prompt medical attention should be sought if a sudden serious hypersensitivity reaction occurs. 3). Benign intracranial hypertension In case of severe or recurrent ataxia, headache, visual problems, nausea and/or vomiting, a funduscopy for papilloedema is recommended.

If papilloedema is confirmed, a diagnosis of benign intracranial hypertension should be considered and, if appropriate, growth hormone treatment should be discontinued. At present there is insufficient evidence to give specific advice on the continuation of growth hormone treatment in patients with resolved intracranial hypertension.

4). 4). Intracranial tumours must be inactive and anti-tumour therapy must be completed prior to starting growth hormone therapy. Treatment should be discontinued if there is evidence of tumour growth. 4). Lonapegsomatropin must not be used for growth promotion in children with closed epiphyses.