'Reimbursement of Pfizer's weekly growth hormone drug offers new hope for adolescent patients'

A local doctor expressed his enthusiasm that the reimbursement of Pfizer Korea's Ngenla (ingredient: Somatropin), a once-weekly growth hormone drug, will enhance compliance among adolescent patients.

At a press conference held to mark the reimbursement at the Koreana Hotel in Seoul on Thursday, Professor Chae Hyeon-uk of the Pediatric Department at Gangnam Severance Hospital spoke on the topic of "Medical unmet needs for pediatric growth hormone deficiency and clinical significance of Ngenla."

"Children undergoing growth hormone treatment face physical and psychological stresses," Chae said. "Treatment adherence has a significant impact on a child's growth rate. There is evidence that patients who miss one or fewer doses a week tend to have better growth rates compared to those missing three or more doses."

He further introduced clinical trial data for Ngenla, noting its non-inferiority in annual height growth rate compared to daily growth hormone drugs and showcasing similar safety and tolerance profiles.

"Between April 2017 and August 2019, a Phase 3 open-label randomized multi-center clinical study was conducted across 21 countries, including Korea," Chae said. "It involved 224 pre-pubertal children with growth hormone deficiencies who were randomly assigned to receive either weekly Ngenla doses (0.66 mg/kg/week) or daily somatropin doses (0.034 mg/kg/day)."

At the 12-month mark, the annual height growth rate for the Ngenla group was 10.10cm while the daily growth hormone group grew at 9.78cm annually, showcasing a difference of 0.33cm, he added.

The professor emphasized that in phase 3 crossover studies, Ngenla's weekly dosing and prefilled pen convenience have been demonstrated to reduce treatment burdens.

"It's preferred by both patients and guardians, enhancing treatment satisfaction," he said. "With its inclusion in insurance coverage, I foresee a shift in the treatment paradigm for growth hormone deficiencies centered on Ngenla."

Kim Hee-jung, executive director of the rare disease division at Pfizer Korea, also said, "We're pleased that Ngenla's reimbursement will broaden access to treatment for children needing growth hormone therapy in Korea."

The company hopes Ngenla, being a weekly dosage, will alleviate the daily treatment burdens faced by children and their guardians, ensuring continuity of care, Kim added.

Ngenla, a recombinant human growth hormone produced through DNA recombination technology with an extended half-life, received approval from the Ministry of Food and Drug Safety on Jan. 31 this year. It is indicated for the treatment of growth failure in children aged three and older due to pituitary growth hormone secretion disorders.

As of Sept. 1, Ngenla is on the reimbursement list, applicable to pediatric patients who meet specific criteria.

Reimbursement eligibility extends to pediatric patients aged three or older with growth hormone secretion disorders, whose height falls at or below the third percentile for their respective age group. It also requires confirmation of diagnosis through two or more growth hormone-inducing tests, and the patient's bone age should be less than the applicable age group.

Treatment coverage begins at the age of three and continues until bone closure or until the bone age reaches 14-15 for females and 15-16 for males. However, patients exceeding a height of 153 centimeters for females and 165 centimeters for males will be responsible for the full cost of treatment.