Adult

The effects of STRENSIQ were studied in adults with pediatric-onset hypophosphatasia (HPP) over 5 years1,2

See the results below from one adult patient’s 6-minute walk test (6MWT) after taking STRENSIQ.

All patient images are hypothetical.
Adult male enjoying the outdoors
Change in plasma PPi Change in plasma PLP 6-minute walk test Bone mineralization
INFANT/YOUNG CHILD INFANT/YOUNG CHILD PEDIATRICADOLESCENT ADOLESCENTADULT
Study 11-3ENB-002-08/
ENB-003-08		 Study 21,5ENB-010-10 Study 31,5,6ENB-006-09/
ENB-008-10		 Study 47ENB-009-10
Design Prospective, single-arm trial and extension in patients with severe perinatal/infantile-onset HPP Prospective, open-label trial in patients with perinatal/infantile-onset HPP Prospective, open-label trial and extension in patients with juvenile- or perinatal/infantile-onset HPP Multicenter, randomized, open-label concurrent control trial including adolescents and adults with pediatric-onset HPP
Duration 7 years 6 years 7 years 5 years
Primary treatment phase 6 months 1-6 years 6 months 6 months
Extension 6.5 years 1-6 years 6.5 years 4.5 years
Number of patients 11 69 13(9 reached adolescencea
during the study8)		 19(13 adults,b
6 adolescents)a
Age at inclusion ≤3 years ≤5 years 6 to 12 years 13 to 65 years

Study 11-3ENB-002-08/ENB-003-08

INFANT/YOUNG CHILD
DESIGN

Prospective, single-arm trial and extension in patients with severe perinatal/infantile-onset HPP

DURATION

7 years

PRIMARY Tx PHASE

6 months

EXTENSION

6.5 years

# OF PATIENTS

11

AGE AT INCLUSION

≤3 years

Study 21,5ENB-010-10

INFANT/YOUNG CHILD
DESIGN

Prospective, open-label trial in patients with perinatal/infantile-onset HPP

DURATION

6 years

PRIMARY Tx PHASE

1-6 years

EXTENSION

—

# OF PATIENTS

69

AGE AT INCLUSION

≤5 years

Study 31,5,61,2,61,4,6ENB-006-09/ENB-008-10

PEDIATRICADOLESCENT
DESIGN

Prospective, open-label trial and extension in patients with juvenile- or perinatal/infantile-onset HPPa

DURATION

7 years

PRIMARY Tx PHASE

6 months

EXTENSION

6.5 years

# OF PATIENTS

13

(9 reached adolescenceab
during the study382)
AGE AT INCLUSION

6 to 12 years

Study 417ENB-009-10

ADOLESCENTADULT
DESIGN

Multicenter, randomized, open-label concurrent control trial including adolescents and adults with pediatric-onset HPPa

DURATION

5 years

PRIMARY Tx PHASE

6 months

EXTENSION

4.5 years

# OF PATIENTS

19

(13 adults,b
6 adolescents)a
AGE AT INCLUSION

13 to 65 years

Abbreviation: HPP, hypophosphatasia.

aAdolescents in the study were aged 13 to 17 years.6-8

bAdults in the study were aged ≥18 years and 12/13 had pediatric-onset HPP.7

aAdolescents in the study were aged 13 to 17 years.2,3

aThe data represented are from subgroup analysis.2

bAdolescents in the study were aged 13 to 17 years.1,2,81,2

aData represent subgroup analysis of adults with pediatric-onset HPP.

bAdults in the study were aged ≥18 years and 12/13 had pediatric-onset HPP.1

Change in plasma PPi (coprimary endpoint)

Change in inorganic pyrophosphate (PPi), a known inhibitor of bone mineralization, from baseline through 5 years1,2

  • One of the coprimary endpoints of this study, change in PPi at 6 months, did not reach statistical significance when comparing the control group to the treatment group. For the total cohort, the within-subject changes in both pyridoxal 5′-phosphate (PLP) and PPi after 6 months and over 5 years of treatment were significant1
  • In adults treated with STRENSIQ (n=12), PPi concentrations were 61% lower after 5 years of treatment (median at baseline: 5.585 µM; median at 5 years: 2.225 µM)2
  • The data shown here are from the open-label extension phase of the study. The study included 19 adult and adolescent patients. Of the 13 adult patients, 12 had pediatric-onset HPP. Three adults were part of the untreated group during the primary phase1
Change in plasma PPi from baseline through 5 years
Change in plasma PPi from baseline through 5 years

Baseline was the last assessment before the first dose of STRENSIQ. The control group began treatment 6 months after the treated group.1

PPi accumulation suppresses hydroxyapatite production, which is necessary for bone mineralization.3

Change in plasma PLP (coprimary endpoint)

Change in PLP, the active form of vitamin B6, from baseline through 5 years1,2

  • The data shown here are from the open-label extension phase of the study. The study included 19 adult and adolescent patients. Of the 13 adult patients, 12 had pediatric-onset HPP. Three adults were part of the untreated group during the primary phase1
  • Over the initial duration, the study used lower doses of STRENSIQ than those in the current FDA-approved label1,4
Change in PLP from baseline through 5 years
Change in PLP from baseline through 5 years

6-minute walk test (secondary endpoint)

Mobility as measured by the 6MWT for adults treated with STRENSIQ2

WATCH 6-MINUTE WALK VIDEO
Mobility as measured by the 6MWT for adult patients treated with Strensiq®
Mobility as measured by the 6MWT for adult patients treated with Strensiq®
  • Improvement in 6MWT was observed as early as 6 months (P=0.0444, treatment vs control groups) and through 5 years. Inferences regarding clinical benefit should not be made2
  • Patients walked a median (min, max) of 324.5 meters (13, 540) at baseline (n=12) vs 429.5 meters (280, 696) at 5 years (n=10)2
    • For adult patients with HPP, the minimal clinically important difference (MCID) is estimated at 23 meters and 31 meters based on 2 distribution-based methods5

  • The data shown here are from the open-label extension phase of the study. The study included 19 adult and adolescent patients. Of the 13 adult patients, 12 had pediatric-onset HPP. Three adults were part of the untreated group during the primary phase1
  • The efficacy results presented here reflect data for adults with pediatric-onset HPP who had available 6MWT data2
  • Over the initial duration, the study used lower doses of STRENSIQ than those in the current FDA-approved label1,4
  • One of the coprimary endpoints of this study, change in PPi at 6 months for the entire study population, did not reach statistical significance1

Bone mineralization (secondary endpoint)

Time to bone mineralization with STRENSIQ1,2

  • The efficacy results presented for bone mineralization include data for 6 patients who had values at baseline and 7 patients with values at 1 year2
  • Reductions in mineralization lag time indicate improved bone mineralization6
  • Mineralization lag time was assessed by bone biopsy at baseline and at 1 year in patients receiving STRENSIQ1
Time to bone mineralization with Strensiq®
Time to bone mineralization with Strensiq®

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Important Safety Information and Indication for STRENSIQ® (asfotase alfa) 40mg/mL vial
Important Safety Information
WARNINGS AND PRECAUTIONS
  • Hypersensitivity Reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and have been observed more than 1 year after treatment initiation. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia.
    Inform patients and/or caregivers of the signs and symptoms of hypersensitivity reactions and have them seek immediate medical care should signs and symptoms occur. If a severe hypersensitivity reaction occurs, discontinue STRENSIQ treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.
  • Lipodystrophy: Localized lipodystrophy, including lipoatrophy (depression in the skin) and lipohypertrophy (enlargement or thickening of tissue), has been reported at injection sites after several months in patients treated with STRENSIQ in clinical trials. Advise patients to follow proper injection technique and to rotate injection sites.
  • Ectopic Calcifications: Patients with HPP are at increased risk for developing ectopic calcifications. Events of ectopic calcification, including ophthalmic (conjunctival and corneal) and renal (nephrocalcinosis, nephrolithiasis), have been reported in the clinical trial experience with STRENSIQ. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported resulting from the occurrence of ectopic calcifications.
    Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function.
  • Possible Immune-Mediated Clinical Effects: In clinical trials, most STRENSIQ-treated patients developed anti-asfotase alfa antibodies and neutralizing antibodies which resulted in reduced systemic exposure of asfotase alfa. In postmarketing reports, some STRENSIQ-treated patients with initial therapeutic response subsequently developed recurrence and worsening in disease-associated laboratory and radiographic biomarkers (some in association with neutralizing antibodies) suggesting possible immune-mediated effects on STRENSIQ’s pharmacologic action resulting in disease progression. The effect of anti-asfotase alfa antibody formation on the long-term efficacy of STRENSIQ is unknown. There are no marketed anti-asfotase alfa antibody tests. If patients experience progression of HPP symptoms or worsening of disease-associated laboratory and imaging biomarkers after a period of initial therapeutic response to STRENSIQ, consider obtaining anti-asfotase alfa antibody testing by contacting STRENSIQ Medical Information at Alexion at 1-888-765-4747 or by email at medinfo@alexion.com. Close clinical follow up is recommended.
ADVERSE REACTIONS
  • Overall, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%). Other common adverse reactions included lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ.
DRUG INTERACTIONS
    Drug Interference with Laboratory Tests:
  • Laboratory tests utilizing alkaline phosphatase (ALP) as a detection reagent could result in erroneous test results for patients receiving treatment due to the presence of asfotase alfa in clinical laboratory samples. Inform laboratory personnel that the patient is being treated with STRENSIQ and discuss use of an alternative testing platform which does not utilize an ALP-conjugated test system.
  • Elevated serum ALP measurements detected through clinical laboratory testing are expected in patients receiving STRENSIQ due to circulating concentrations of asfotase alfa and may be unreliable for clinical decision making.
SPECIAL POPULATIONS
  • Pregnancy & Lactation: There are no available data on STRENSIQ use in pregnant women, the presence of STRENSIQ in human milk, or the effects on the breastfed infant or on milk production, to inform a drug associated risk.
Indication

STRENSIQ® is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

Please see STRENSIQ (asfotase alfa) full Prescribing Information.
Important Safety Information and Indication for STRENSIQ® (asfotase alfa) 40mg/mL vial
Important Safety Information
WARNINGS AND PRECAUTIONS
  • Hypersensitivity Reactions, including anaphylaxis, have been reported in STRENSIQ-treated patients. Signs and symptoms consistent with anaphylaxis included difficulty breathing, choking sensation, nausea, periorbital edema, and dizziness. These reactions have occurred within minutes after subcutaneous administration of STRENSIQ and have been observed more than 1 year after treatment initiation. Other hypersensitivity reactions have also been reported in STRENSIQ-treated patients, including vomiting, fever, headache, flushing, irritability, chills, skin erythema, rash, pruritus and oral hypoesthesia.
    Inform patients and/or caregivers of the signs and symptoms of hypersensitivity reactions and have them seek immediate medical care should signs and symptoms occur. If a severe hypersensitivity reaction occurs, discontinue STRENSIQ treatment and initiate appropriate medical treatment. Consider the risks and benefits of re-administering STRENSIQ to individual patients following a severe reaction. If the decision is made to re-administer the product, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.
  • Lipodystrophy: Localized lipodystrophy, including lipoatrophy (depression in the skin) and lipohypertrophy (enlargement or thickening of tissue), has been reported at injection sites after several months in patients treated with STRENSIQ in clinical trials. Advise patients to follow proper injection technique and to rotate injection sites.
  • Ectopic Calcifications: Patients with HPP are at increased risk for developing ectopic calcifications. Events of ectopic calcification, including ophthalmic (conjunctival and corneal) and renal (nephrocalcinosis, nephrolithiasis), have been reported in the clinical trial experience with STRENSIQ. There was insufficient information to determine whether or not the reported events were consistent with the disease or due to STRENSIQ. No visual changes or changes in renal function were reported resulting from the occurrence of ectopic calcifications.
    Ophthalmology examinations and renal ultrasounds are recommended at baseline and periodically during treatment with STRENSIQ to monitor for signs and symptoms of ophthalmic and renal ectopic calcifications and for changes in vision or renal function.
  • Possible Immune-Mediated Clinical Effects: In clinical trials, most STRENSIQ-treated patients developed anti-asfotase alfa antibodies and neutralizing antibodies which resulted in reduced systemic exposure of asfotase alfa. In postmarketing reports, some STRENSIQ-treated patients with initial therapeutic response subsequently developed recurrence and worsening in disease-associated laboratory and radiographic biomarkers (some in association with neutralizing antibodies) suggesting possible immune-mediated effects on STRENSIQ’s pharmacologic action resulting in disease progression. The effect of anti-asfotase alfa antibody formation on the long-term efficacy of STRENSIQ is unknown. There are no marketed anti-asfotase alfa antibody tests. If patients experience progression of HPP symptoms or worsening of disease-associated laboratory and imaging biomarkers after a period of initial therapeutic response to STRENSIQ, consider obtaining anti-asfotase alfa antibody testing by contacting STRENSIQ Medical Information at Alexion at 1-888-765-4747 or by email at medinfo@alexion.com. Close clinical follow up is recommended.
ADVERSE REACTIONS
  • Overall, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%). Other common adverse reactions included lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ.
DRUG INTERACTIONS
    Drug Interference with Laboratory Tests:
  • Laboratory tests utilizing alkaline phosphatase (ALP) as a detection reagent could result in erroneous test results for patients receiving treatment due to the presence of asfotase alfa in clinical laboratory samples. Inform laboratory personnel that the patient is being treated with STRENSIQ and discuss use of an alternative testing platform which does not utilize an ALP-conjugated test system.
  • Elevated serum ALP measurements detected through clinical laboratory testing are expected in patients receiving STRENSIQ due to circulating concentrations of asfotase alfa and may be unreliable for clinical decision making.
SPECIAL POPULATIONS
  • Pregnancy & Lactation: There are no available data on STRENSIQ use in pregnant women, the presence of STRENSIQ in human milk, or the effects on the breastfed infant or on milk production, to inform a drug associated risk.
Indication

STRENSIQ® is indicated for the treatment of patients with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

Please see STRENSIQ (asfotase alfa) full Prescribing Information.
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References

1. Kishnani PS, Rockman-Greenberg C, Rauch F, et al. Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia. Bone. 2019;121:149-162.

2. Data on file. Alexion Pharmaceuticals.

3. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(suppl 2):380-388.

4. STRENSIQ [package insert]. Alexion Pharmaceuticals Inc.

5. Phillips D, Tomazos IC, Moseley S, L’Italien G, Gomes da Silva H, Lerma Lara S. Reliability and validity of the 6-minute walk test in hypophosphatasia. JBMR Plus. 2019;3(6):e10131.

6. Ott SM. Histomorphometric measurements of bone turnover, mineralization, and volume. Clin J Am Soc Nephrol. 2008;3(suppl 3):S151-S156.

7. Recker RR, Kimmel DB, Parfitt AM, Davies KM, Keshawarz N, Hinders S. Static and tetracycline-based bone histomorphometric data from 34 normal postmenopausal females. J Bone Miner Res. 1988;3(2):133-144.

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