dm+d

Unassigned

Refrigerated Storage

Ngenla Pfizer

Pfizer
Ngenla
Solution for injection in pre-filled pen

In the event of an inadvertent temperature excursion the following data may be used:
Ngenla may be held at room temperature (up to 32°C) for up to 4 hours with each injection for a maximum of 5 times. Return Ngenla to the refrigerator again after each use. Discard the pen if it has been exposed to temperatures >32°C or if it has been removed from the refrigerator for more than 4 hours with each use.

Additional data shows the product is stable with no impact on product quality when exposed to 30°C for 7 days.

Please also refer to the manufacturer’s product literature for recommended storage information at https://www.medicines.org.uk

As above
7 October 2022
London MI Service

New Medicines

NgenlaGrowth hormone deficiency in children

Information

Ngenla
New molecular entity
Pfizer
Pfizer

Development and Regulatory status

Launched
Approved (Licensed)
Not approved
May 2022
Yes
Yes
May 22Available in the UK. Price for Ngenla 24mg/1.2ml solution for injection pre-filled pen x 1 = £189.60; 60mg/1.2ml solution for injection pre-filled pen x 1 = £474.00 [16].
Apr 22Granted orphan drug status in UK [15].
Mar 22Approved in the UK for the "treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone." [14].
Feb 22Approved in the EU [13]
Jan 22US FDA has declined approval and issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for somatrogon for the treatment of growth hormone deficiency (GHD) in paediatric patients. Pfizer is evaluating the FDA’s comments and will work with the agency to determine an appropriate path forward. The decision was unexpected considering Ngenla has approval in Canada, Japan and Australia and a positive opinion from the EMA [12].
Dec 21Recommended for EU approval by CHMP “for the treatment of children and adolescents from 3 years of age with growth disturbance due to insufficient secretion of growth hormone.” Ngenla will be available as 24 mg/1.2 ml and 60 mg/1.2 ml solutions for injection [11].
Sep 21FDA decision delayed with PUFDA date delayed till January 22 [10].
Feb 21EMA has accepted for review the MAA for somatrogon for once-weekly treatment of pediatric patients with growth hormone deficiency. Pfizer expects a decision from the EC in 2022 [9].
Jan 21Pfizer have exclusive licence to commercialise somatrogon [8].
Jan 21Filed in US, with decision anticipated by October 21 [7].
Sep 19Company now OPKO Health [4].
Feb 18Has orphan drug status in EU & US [2]

Category

A long-acting, proprietary formulation of somatropin, for once weekly or bi-monthly administration. Disposable pre-filled pen.
Growth-hormone deficiency affected approximately 4 in 10,000 people in the European Union (EU) [1].
Growth hormone deficiency in children
Subcutaneous injection

Further information

Yes

Trial or other data

Oct 20PIII open-label, crossover study evaluating somatrogon dosed once-weekly in children aged 3 to <18 years with growth hormone deficiency met its primary endpoint of improved treatment burden compared to somatropin (GENOTROPIN) administered once-daily after 12 weeks of treatment. Mean overall Life Interference total score with somatrogon was 8.63 vs. 24.13 [6].
Oct 19OPKO and Pfizer announce positive phase III data for somatrogon in treatment of pre-pubertal children with growth hormone deficiency; in study (n=224; NCT02968004), treatment with somatrogon was non-inferior to somatropin dosed once-daily with respect to height velocity at 12 months of treatment (primary endpoint;10.12 vs. 9.78 cm/year, respectively) [5].
Sep 19PIII trial comparing once-weekly somatrogon vs. once-daily Genotropin® (somatropin) has completed, topline results expected Q4 2019 [4].
Dec 17PIII (NCT02968004) study is recruiting its. Collection of primary outcome at data (annual height velocity in cm/year measured after 12 months of treatment) is due to complete Jun 19 [3].
Dec 16OPKO Health initiates a pivotal PIII trial to evaluate a weekly single dose of somatropin long acting (delivered in a pen device) versus daily injections of growth hormone injection in paediatric pre-pubertal growth hormone deficient patients (NCT02968004; CP4-006). The open-label, parallel, prospective, randomised trial is enrolling 220 patients in the US [2].

Evidence based evaluations

NgenlaGrowth hormone deficiency in adults - long-acting formulation of somatotropin

Information

Ngenla
Licence extension / variation
Pfizer
Pfizer

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

A long-acting, proprietary formulation of somatropin, for once weekly or bi-monthly administration. Disposable pre-filled pen. The technology used to develop long-acting somatropin is based on the carboxyl terminal peptide (CTP) that has been shown to extend the active time of therapeutic proteins in the body without increasing toxicity or reducing biological activity.
Growth-hormone deficiency affected approximately 4 in 10,000 people in the European Union (EU) [3].
Growth hormone deficiency in adults - long-acting formulation of somatotropin
Subcutaneous