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Long-Acting Therapies Will Expand Growth Hormone Deficiency Market

Kunj Gohil PharmD, RPh

Growth hormone deficiency (GHD) results from impaired secretion of growth hormone (GH) from the pituitary gland.1 Typically, the disease is classified in one of three categories: congenital, acquired, or idiopathic. Congenital GHD is present at birth and can be caused either by genetic mutations or structural abnormality in the brain. Acquired GHD occurs later in life as a result of an event that affects the brain, such as trauma, infection, or tumor, among many other causes. Finally, idiopathic GHD has an unknown or undiagnosed cause.2

Childhood GHD can often be made up of all three categories and can have developmental consequences as the child matures. Current treatment for GHD consists of patient-specific GH replacement therapy.2 Pediatric GH replacement therapy is given in a calculated weight-based dosing regimen, whereas adult dosing can be either weight-based or non-weight-based.3 Several somatropin, recombinant human GH brands are available.2 Looking to the future, therapies in the pipeline are expected to include long-acting formulations.4 It is anticipated that these formulations will increase the use of and improve adherence to the medications. The entrance of several long-acting GH agents is expected to translate to an increase in global sales from $1.26 billion in 2014 to $1.88 billion in 2024.4

Future Therapies

Drug
Manufacturer
Status Regimen Information Expected Approval Anticipated Peak Year Sales/Pricing
Somatropin Biopartners
LG Life Sciences
Phase 3 2 mg per week starting dose via SC injection 2017 Priced slightly lower than other long-acting GH drugs, at a 15–20% premium above Genotropin
NNC0195-0092
Novo Nordisk
Phase 3 0.02–0.08 mg/kg per week 2018 Priced at a premium of 20% above Norditropin
VRS-317
Versartis Inc.
Phase 3 1.15 mg/kg per week, or 2.5 mg/kg twice monthly, or 5 mg/kg monthly 2018 Priced higher than competitors and will command a premium of 25% above Genotropin
MOD-4023
OPKO Health
Phase 2/3 0.02–0.08 mg/kg per week 2017 Priced at a premium of 20% above Genotropin
ACP-001
Ascendis Pharma
Phase 2 0.02–0.08 mg/kg per week 2020 Priced at a premium that has been assumed to be 18% above Genotropin
TV-1106
Teva Pharmaceuticals
Phase 2 0.02–0.08 mg/kg per week 2019 Priced at a premium that has been assumed to be 15% above Genotropin

SC = subcutaneous

Sources: FDA; GlobalData; company websites; ClinicalTrials.gov

Current Therapiesa

Drug
Manufacturer
Approval Date Indicationb Regimen Informationc
Omnitrope biosimilar
Sandoz
May 30, 2006 GHD, SGA, PWS, TS, ISS 0.16 to 0.24 mg/kg per week
Saizen
Merck Serono
October 8, 1996 GHD 0.18 mg/kg per week
Genotropin
Pfizer
August 24, 1995 GHD, SGA, PWS, TS, ISS 0.16 to 0.24 mg/kg per week
Zomacton
Ferring Pharmaceuticals
May 25, 1995 GHD 0.3 mg/kg per week
Norditropin
Novo Nordisk
May 8, 1995 GHD, SGA, TS, NS 0.024 to 0.034 mg/kg per day
Nutropin AQ
Roche
November 17, 1993 GHD, TS, ISS, GFCKD 0.3 mg/kg per week
Humatrope
Lilly
March 8, 1987 GHD, SGA, TS, ISS, SHOX-D 0.18 to 0.30 mg/kg per week

aThis list is not all-inclusive; additional therapies may be available for this condition. Pricing is not included in this article due to variability in dosing and formulations for each agent.

bAbbreviated indication provided; for full indication, please refer to prescribing information.

cRegimens based on the recommended dosage and maintenance phases from prescribing information for pediatric patients; typical doses and titration schedules may vary based on patient-specific requirements.

GFCKD = growth failure secondary to chronic kidney disease; GHD = growth hormone deficiency; ISS = idiopathic short stature; NS = Noonan syndrome; PWS = Prader-Willi syndrome; SGA = small for gestational age; SHOX-D = short stature homeobox-containing gene deficiency; TS = Turner syndrome

Sources: Drugs@FDA and prescribing information for all medications

Author bio: 
Dr. Gohil is Central Services Manager with Medical Services at MediMedia Managed Markets in Yardley, Pennsylvania.

References

  1. U.S. NationalLibrar y of Medicine Medline Plus. Growth hormone deficiency—children. May 142014;Available at: https://medlineplus.gov/ency/article/001176.htm. Accessed October 18, 2015
  2. National Organization for Rare Disorders. Growth hormone deficiency. 2012;Available at: https://rarediseases.org/rare-diseases/growth-hormone-deficiency/. Accessed October 18, 2015
  3. Loguidice C. Pediatric growth hormone deficiency: drugs in the pipeline. Rare Disease Report February 92015;Available at: https://www.mdmag.com/medical-news/pediatric-growth-hormone-deficiency-pipeline. Accessed October 18, 2015
  4. GlobalData. OpportunityAnalyzer: growth hormone deficiency—opportunity analysis and forecast to 2024. August 2015;Available at: https://www.globaldata.com/store/report/GDHC029POA--/?. Accessed October 18, 2015