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FDA committee recommends new indication to treat short children
In addition, FDA approves a new low-dose strength of conjugated estrogens/medroxyprogesterone acetate tablets.
By Jay Lewis
Staff Writer
July 2003
BETHESDA, Md. – At the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting in June, the committee recommended approval of a new indication for recombinant growth hormone therapy (Humatroper, Eli Lilly).
The recombinant growth hormone therapy is currently approved only for use in pediatric and adult patients who have growth hormone deficiencies, as well as pediatric patients with Turner’s syndrome.
The new proposed use for the recombinant growth hormone therapy is for the long-term treatment of non-growth hormone deficient short stature. This condition is defined by height standard deviation score less than or equal to –2.25 in pediatric patients whose epiphyses are not closed and in whom diagnostic evaluation excludes causes of short stature.
The committee voted 8-2 in favor of approving recombinant growth hormone therapy for use in non-growth hormone deficient short stature. The EMDAC members based their recommendations for approval on the results of a pivotal, double-blind, randomized, placebo-controlled trial in 71 patients. This study followed patients on recombinant growth hormone therapy from childhood until adulthood and found that the treatment can make children with non-growth hormone deficient short stature grow 1.5 inches to 2.8 inches taller than expected.
If the FDA follows the EMDAC recommendation and approves this treatment, Lilly will be the first growth hormone maker allowed to market a drug for abnormally short but otherwise healthy children. The proposed treatment requirements are for pediatric patients who are predicted to be well below average in height as adults — which is a height of 5 feet 3 inches for boys and 4 feet 11 inches for girls.
Difficult decision
Although most of the committee members voted for recommending approval, several members of the panel had some concerns with the treatment.
The new proposed use for the recombinant growth hormone therapy is for the
long-term treatment of non-growth hormone deficient short stature.
Several panelists questioned whether the small improvement in growth expected with the recombinant therapy would help children’s overall quality of life enough to justify the costs and the challenges of the treatment. The treatment involves daily injections and costs are estimated at $10,000 to $25,000 per year.
There were also ethical concerns, as committee members expressed unease with a treatment that was designed only to increase height, even though the patients were healthy in all other respects. Furthermore, the committee was concerned about the proper age to begin treatment and its overall efficacy.
The panelists finally decided to recommend approval, but with the condition that Eli Lilly does additional research on how best to use the drug. Eli Lilly would also be required to start a mandatory registry to track every child who was prescribed the recombinant growth hormone therapy.
Based on the EMDAC recommendations, Eli Lilly pledged tight restrictions on the drug’s availability. The company said that if FDA approves the new use, the drug would be prescribed only by endocrinologists and shipped by specially designated pharmacies to patients. Patients would also be required to undergo several growth tests.
In related news, the FDA recently approved a new low-dose strength of conjugated estrogens/medroxyprogesterone tablets (Prempro, Wyeth). The new low-dose tablets contain 0.3 mg of conjugated estrogens and 1.5 mg of medroxyprogesterone.
These new low-dose tablets were approved for the prevention of postmenopausal osteoporosis, as well as for the treatment of moderate to severe symptoms associated with menopause, including hot flashes, night sweats and vaginal dryness.
The FDA also expanded the approved uses for Prempro tablets in the dose of 0.45 mg/1.5 mg to include the prevention of postmenopausal osteoporosis.
Wyeth said it expects to have Prempro 0.45 mg/1.5 mg available in early summer 2003, and Prempro 0.3 mg/1.5 mg available in late 2003.
“These approvals mean that women and clinicians will have two low-dose options to better individualize effective treatment for menopausal symptom relief and concomitant bone protection,” said Victoria Kusiak, vice president global medical affairs and North American medical director of Wyeth Pharmaceuticals.
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FDA committee recommends new indication to treat short children
In addition, FDA approves a new low-dose strength of conjugated estrogens/medroxyprogesterone acetate tablets.
By Jay Lewis
Staff Writer
July 2003
BETHESDA, Md. – At the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting in June, the committee recommended approval of a new indication for recombinant growth hormone therapy (Humatroper, Eli Lilly).
The recombinant growth hormone therapy is currently approved only for use in pediatric and adult patients who have growth hormone deficiencies, as well as pediatric patients with Turner’s syndrome.
The new proposed use for the recombinant growth hormone therapy is for the long-term treatment of non-growth hormone deficient short stature. This condition is defined by height standard deviation score less than or equal to –2.25 in pediatric patients whose epiphyses are not closed and in whom diagnostic evaluation excludes causes of short stature.
The committee voted 8-2 in favor of approving recombinant growth hormone therapy for use in non-growth hormone deficient short stature. The EMDAC members based their recommendations for approval on the results of a pivotal, double-blind, randomized, placebo-controlled trial in 71 patients. This study followed patients on recombinant growth hormone therapy from childhood until adulthood and found that the treatment can make children with non-growth hormone deficient short stature grow 1.5 inches to 2.8 inches taller than expected.
If the FDA follows the EMDAC recommendation and approves this treatment, Lilly will be the first growth hormone maker allowed to market a drug for abnormally short but otherwise healthy children. The proposed treatment requirements are for pediatric patients who are predicted to be well below average in height as adults — which is a height of 5 feet 3 inches for boys and 4 feet 11 inches for girls.
Difficult decision
Although most of the committee members voted for recommending approval, several members of the panel had some concerns with the treatment.
The new proposed use for the recombinant growth hormone therapy is for the
long-term treatment of non-growth hormone deficient short stature.
Several panelists questioned whether the small improvement in growth expected with the recombinant therapy would help children’s overall quality of life enough to justify the costs and the challenges of the treatment. The treatment involves daily injections and costs are estimated at $10,000 to $25,000 per year.
There were also ethical concerns, as committee members expressed unease with a treatment that was designed only to increase height, even though the patients were healthy in all other respects. Furthermore, the committee was concerned about the proper age to begin treatment and its overall efficacy.
The panelists finally decided to recommend approval, but with the condition that Eli Lilly does additional research on how best to use the drug. Eli Lilly would also be required to start a mandatory registry to track every child who was prescribed the recombinant growth hormone therapy.
Based on the EMDAC recommendations, Eli Lilly pledged tight restrictions on the drug’s availability. The company said that if FDA approves the new use, the drug would be prescribed only by endocrinologists and shipped by specially designated pharmacies to patients. Patients would also be required to undergo several growth tests.
In related news, the FDA recently approved a new low-dose strength of conjugated estrogens/medroxyprogesterone tablets (Prempro, Wyeth). The new low-dose tablets contain 0.3 mg of conjugated estrogens and 1.5 mg of medroxyprogesterone.
These new low-dose tablets were approved for the prevention of postmenopausal osteoporosis, as well as for the treatment of moderate to severe symptoms associated with menopause, including hot flashes, night sweats and vaginal dryness.
The FDA also expanded the approved uses for Prempro tablets in the dose of 0.45 mg/1.5 mg to include the prevention of postmenopausal osteoporosis.
Wyeth said it expects to have Prempro 0.45 mg/1.5 mg available in early summer 2003, and Prempro 0.3 mg/1.5 mg available in late 2003.
“These approvals mean that women and clinicians will have two low-dose options to better individualize effective treatment for menopausal symptom relief and concomitant bone protection,” said Victoria Kusiak, vice president global medical affairs and North American medical director of Wyeth Pharmaceuticals.
[Endocrine Today Homepage]
[Current Issue] [Back Issues]
[Endocrine News] [Practice Management]
[Meetings & Courses] [Industry Link]
[Professional Marketplace]
__________________
