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Learn more about Strensiq® (asfotase alfa) and HPP.

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Cases of patients with perinatal/infantile- or juvenile-onset hypophosphatasia (HPP) treated with Strensiq®

Click to explore patient cases

Strensiq® improved skeletal manifestations, healing of rachitic chest and respiratory function, and survival.1,10,a

Study 1  |  Patient: 7.5-month-old female infant15  |  Diagnosis: Perinatal-onset hypophosphatasia15

Patient 002-01-01: Published as patient 1 in Whyte MP, Greenberg CR, Salman N, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia [supplementary appendix]. N Engl J Med. 2012;366:(10)S1-S47.

Hypophosphatasia history15

  • Prenatal suspicion of HPP: Bowed and angulated extremities on ultrasound
  • Diagnosis of perinatal-onset HPP confirmed at birth: Low alkaline phosphatase and radiographic evidence of long bone bowing and minimal rachitic changes
  • 5 months of age: Failure to thrive, hypercalcemia, feeding difficulties, vomiting, respiratory insufficiency, and worsening skeletal disease with nontraumatic fractures

Morbidities at baseline (7.5 months)15

  • Skeletal: Demineralization, severe rachitic changes, large anterior fontanelle, short and bowed limbs, scoliosis
  • Muscular: Hypotonia, gross motor delay (minimal head control), fed by nasogastric tube
  • Respiratory support: Noninvasive ventilator support
  • Hypercalcemia and nephrocalcinosis: Treated with a low-calcium diet
  • Low alkaline phosphatase: 8 U/L; age- and gender-adjusted lower limit of normal: 117 U/L

RGI-C scale

Hypophosphatasia-related rickets was assessed using the RGI-C scale.1,14

Radiographic Global Impression of Change (RGI-C) Scale1,14

 

The RGI-C scale evaluates change in paired radiographs of the chest and bilateral wrists and knees14

Scores measure change from baseline at prespecified time points14

aSee the Clinical Data section of this website for additional data from clinical trials in infants with perinatal/infantile-onset HPP.

Treatment with Strensiq® resulted in skeletal, mobility, and growth improvements.1,11,a

Study 3  |  Patient: 6-year-old male child16  |  Diagnosis: Juvenile-onset hypophosphatasia16

Patient 006-01-08: Published as patient 7 in Whyte MP, Madson KL, Phillips D, et al. Asfotase alfa therapy for children with hypophosphatasia [supplementary appendix]. JCI Insight. 2016;1(9):e85971.

Hypophosphatasia history16

  • 13 months of age: Knock-kneed stance
  • 14 months of age: Tooth loss
  • 18 months of age: Rickets with metaphyseal flaring at wrists
  • 2.5 years of age: Confirmed diagnosis of juvenile-onset HPP
  • At 5 years of age: Had difficulty walking

Morbidities at baseline (6 years)16

  • Growth: Slightly below average height and weight
    (z scores: -0.9 and -1.3, respectively)
  • Low alkaline phosphatase: 48 U/L; age- and gender-adjusted lower limit of normal: 93 U/L

RGI-C scale

Hypophosphatasia-related rickets was assessed using the RGI-C scale.1,14

Radiographic Global Impression of Change (RGI-C) Scale1,14

 

The RGI-C scale evaluates change in paired radiographs of the chest and bilateral wrists and knees14

Scores measure change from baseline at prespecified time points14

aSee the Clinical Data section of this website for additional data from clinical trials in children and adolescents with juvenile-onset HPP.

Treatment with Strensiq® improved skeletal manifestations and mobility.1,11,a

Study 3  |  Patient: 12-year-old male adolescent16  |  Diagnosis: Juvenile-onset hypophosphatasia16

Patient 006-01-06: Published as patient 5 in Whyte MP, Madson KL, Phillips D, et al. Asfotase alfa therapy for children with hypophosphatasia [supplementary appendix]. JCI Insight. 2016;1(9):e85971.

Hypophosphatasia history16

  • 1 to 2 years of age: Failure to thrive, delayed gross motor skills, and premature tooth loss
  • 3.5 years of age: Confirmed diagnosis of juvenile-onset hypophosphatasia
  • By 5 years of age: Had lost 7 teeth

Morbidities at baseline (12 years)16

  • Systemic manifestations: Knock-knees, ectopic calcification (eye)
  • Functional abnormalities: Walked with unusual gait, could not keep up with peers
  • Growth: Height and weight z scores were -1.3 and -0.1, respectively
  • Low alkaline phosphatase: 35 U/L; age- and gender-adjusted lower limit of normal: 95 U/L

RGI-C scale

Hypophosphatasia-related rickets was assessed using the RGI-C scale.1,14

Radiographic Global Impression of Change (RGI-C) Scale1,14

 

The RGI-C scale evaluates change in paired radiographs of the chest and bilateral wrists and knees14

Scores measure change from baseline at prespecified time points14

aSee the Clinical Data section of this website for additional data from clinical trials in children and adolescents with juvenile-onset HPP.

Strensiq® improved mobility.9,a

Study 4  |  Patient: 57-year-old female adult9  |  Diagnosis: Juvenile-onset hypophosphatasia9

Patient 009-03-07: Publication in development.

Hypophosphatasia history9

  • Confirmed diagnosis of juvenile-onset hypophosphatasia

Morbidities at baseline (57 years)9

  • Below normal mobility as measured by 6-minute walk test (6MWT)
aSee the Clinical Data section of this website for additional data from a clinical trial in adults with perinatal/infantile- or juvenile-onset HPP.
bBaseline distance includes patients in the control group (n=3) and those treated with Strensiq (n=7) for the first 24 weeks of the study9

Indication

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

Important Safety Information

  • 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 can occur in patients on treatment for more than one year. 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. 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.
  • Localized lipodystrophy, including lipoatrophy and lipohypertrophy, has been reported at injection sites after several months in patients treated with STRENSIQ. Advise patients to follow proper injection technique and to rotate injection sites.
  • Patients with HPP are at increased risk for developing ectopic calcifications. In clinical trials, 14 cases (14%) of ectopic calcification of the eye including the cornea and conjunctiva, and the kidneys (nephrocalcinosis) were reported. 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. 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.
  • The most common adverse reactions (≥ 10%) are injection site reactions, lipodystrophy, ectopic calcifications and hypersensitivity reactions.