STRENSIQ helps build their framework and gets them moving1 IN CHILDREN WITH HYPOPHOSPHATASIA
     
  • HPP-related rickets
  • Mobility
  • Pain/disability
  • Strength/agility
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    CHILDREN
    Study design

    of patients achieved complete or substantial healing of HPP-related rickets as measured by the RGI-C scale*

    of patients achieved complete or substantial healing of HPP-related rickets as measured by the RGI-C scale*

    *The RGI-C scale quantifies change over time in mineralization and bone structure.
    †12 patients entered the long-term extension of the trial.3
    RGI-C score: +2.67
    RGI-C score: +2.67
    RGI-C score: +2.00
    RGI-C score: +2.00
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    A retrospective chart review of the natural history of hypophosphatasia in 32 untreated children with juvenile-onset hypophosphatasia and similar characteristics showed no improvement in height z scores from baseline to last assessment (61 months).6
    • Mean (min, max) height z score at baseline: -1.1 (-4.9, 2.6)7
    • Mean (min, max) height z score at last assessment: -1.1 (-4.9, 1.8)7
    AFTER 6 MONTHS8

    29%
    median change from baseline at 6 months in distance walked (482 meters vs 350 meters) (P=0.001)

    AFTER 7 YEARS8

    68%
    median change from baseline at 7 years in distance walked (588 meters vs 350 meters) (P=0.001)
    For children with hypophosphatasia, the minimal clinically important difference (MCID) is estimated at 21 meters and 31 meters based on 2 distribution-based methods.9

    6MWT: Patients walk an indoor, flat, 30-meter walkway with cones at the beginning and end to indicate turns. At the end of 6 minutes, the total distance is recorded.10

    Representations of efficacy are consistent with results seen from typical patients in the study. Individual results may vary.
    STRENSIQ OPEN-LABEL EXTENSION: PAIN AND DISABILITY
    View CHAQ disability index
    CHAQ discomfort index measured hypophosphatasia-related pain (eg, bone, joint, and muscle pain) and is determined by visual analog scale of pain (0=no pain, 100=very severe pain).2,4,11

    MEDIAN DISCOMFORT INDEX SCORE (PAIN)1,2

    *Not statistically significant.
    CHAQ disability index measures activities of daily living. Scores range from 0-3, with lower scores indicating less disability.2,4,11


    MEDIAN DISABILITY INDEX SCORE (PAIN)2,4


    CHAQ is a patient/caregiver-reported assessment not validated specifically for hypophosphatasia so inferences of clinical benefit should not be made. CHAQ was developed to assess health status for children with arthritis. Interpretation of these findings is limited due to the lack of control group and potential bias introduced by use of parent-reported data for patients. Minimal clinically important difference (MCID) has not been established for the hypophosphatasia population.12,14
    STRENSIQ OPEN-LABEL EXTENSION: STRENGTH AND AGILITY
    Changes in strength and agility scores were observed up to 6 months from baseline through 7 years.
    • At baseline, patients showed lower strength and agility scores than healthy age-matched peers

    BOT-2 measures hand and arm coordination, balance, mobility, and strength relative to healthy same-aged peers. BOT-2 is not validated specifically for hypophosphatasia so inferences of clinical benefit should not be made. It has been validated in pediatric populations (aged 4 to 21 years) with mild to moderate motor impairment.2,4,5,15,16
    Adolescents
    Data on file. Alexion Pharmaceuticals, Inc. Whyte MP, Madson KL, Phillips D, et al. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016;1(9):e85971. Whyte MP, Fujita KP, Moseley S, Thompson DD, McAlister WH. Validation of a novel scoring system for changes in skeletal manifestations of hypophosphatasia in newborns, infants, and children: the radiographic global impression of change scale. J Bone Miner Res. 2018;33(5):868-874. Whyte MP, Rockman-Greenberg C. Moseley S, Denker AE, McAlister WH. Sustained radiographic and functional improvements with asfotase alfa treatment for up to 7 years in children with hypophosphatasia. Oral presentation at: 8th Biennial International Conference on Children’s Bone Health (ICCBH); June 10-13, 2017; Würzburg, Germany. Whyte MP, et al. Abstract presented at: 8th Biennial International Conference on Children’s Bone Health (ICCBH); June 10-13, 2017; Würzburg, Germany. Whyte MP, Madson KL, Munns CF, et al. A retrospective, multi-national, non-interventional, natural history study of the childhood form of hypophosphatasia. Presented at: Endocrine Society’s 97th Annual Meeting and Expo; March 15, 2015; San Diego, CA. STRENSIQ. Package Insert. Alexion Pharmaceuticals, Inc. Data on file. Alexion Pharmaceuticals, Inc. Phillips D, Tomazos IC, Moseley S, et al. Reliability and validity of the 6-minute walk test in hypophosphatasia. JBMR Plus. 2019;3(6):e10131. ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002;166(1):111-117. Dempster H, Porepa M, Young N, Feldman BM. The clinical meaning of functional outcome scores in children with juvenile arthritis. Arthritis Rheum. 2001;44(8):1768-1774. Klepper SE. Measures of pediatric function. Arthritis Rheum. 2003;49(suppl 5):S5-S14. Klepper SE. Measures of pediatric function. Arthritis Care Res. 2011;63(suppl 11):S371-S382. Whyte MP, Rockman-Greenberg C, Moseley S, Denker AE, Watsky E, McAlister WH. Sustained radiographic and functional improvements with asfotase alfa treatment for up to 7 years in children with hypophosphatasia. Poster presented at: International Congress of Inborn Errors of Metabolism (ICIEM); Sept 5–8, 2017; Rio de Janeiro, Brazil. Bruininks RH, Bruininks BD. Pearson. Updated October 22, 2013. Accessed April 4, 2023. http://images.pearsonclinical.com/images/Assets/BOT-2/ BOT-2_Complete_Form_Sample_Report.pdf Bruininks RH, Bruininks BD. Pearson. Accessed April 4, 2023. https://www.pearsonassessments.com/store/usassessments/en/Store/Professional-Assessments/Motor-Sensory/Bruininks-Oseretsky-Test-of-Motor-Proficiency-%7C-Second-Edition/p/100000648.html?tab=product-details
    WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
    Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.

    Initiate STRENSIQ under the supervision of a healthcare provider with appropriate medical monitoring and support measures. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue STRENSIQ and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur [see Warnings and Precautions (5.1)].
    • Life-threatening 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, erythema, rash, pruritus, and oral hypoesthesia. Consider the risks and benefits of re-administering STRENSIQ following a severe reaction. If the decision is made to re-administer, monitor patients for a reoccurrence of signs and symptoms of a severe hypersensitivity reaction.

    • Lipodystrophy: Localized lipodystrophy, including lipoatrophy and lipohypertrophy 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 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.
    In clinical trials, the most common adverse reactions (≥ 10%) reported were injection site reactions (63%), lipodystrophy (28%), ectopic calcifications (14%), and hypersensitivity reactions (12%). Possible immune-mediated clinical effects have been identified during post-approval use of STRENSIQ.
     
    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. Do not rely on serum ALP measurements for clinical decision making in patients treated with STRENSIQ.
    • 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.
    To report SUSPECTED ADVERSE REACTIONS, contact Alexion Pharmaceuticals, Inc. at 1-844-259-6783 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch

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

    Please see full Prescribing Information for STRENSIQ (asfotase alfa), including Boxed WARNING regarding hypersensitivity reactions including anaphylaxis.