HPP REAL STORIES

Meet Evie

Diagnosed with HPP at 2 weeks old

When an ultrasound revealed that Evie had extremely fragile and transparent bones, doctors told expectant parents John and Lindsey that it was not likely their baby girl would live long after birth. The hours that Evie’s parents expected to have with her turned into weeks, but the baby was gripped by seizures.

Evie, diagnosed with HPP at 2 weeks old, playing on a playground

Then, a routine blood draw revealed that Evie had low alkaline phosphatase (ALP), which enabled them to diagnose her with hypophosphatasia (HPP) – an ultra-rare, genetic, potentially life-threatening metabolic condition that prevents minerals such as calcium and phosphate from being properly deposited in bones.1-4

Evie, diagnosed with HPP at 2 weeks old, riding a toy car Evie, diagnosed with HPP at 2 weeks old, painting

Her parents were told that Evie’s case was very severe and that there were no approved or effective treatment options. At three months old, Evie was enrolled in a clinical trial to receive an investigational targeted enzyme replacement therapy, which was approved in 2015 and is now known as STRENSIQ® (asfotase alfa). Evie continues to receive STRENSIQ today.

Evie is, as her mother says, “writing the book on where we go from here. Her story gives other parents with special needs kids hope that they can be okay, and things can be good.”*

*The results from use of STRENSIQ may vary.


IMPORTANT SAFETY INFORMATION

What is Strensiq® (asfotase alfa)?
Strensiq is a prescription medicine used to treat people with perinatal/infantile- and juvenile-onset hypophosphatasia (HPP).

What are the possible side effects of Strensiq?
Strensiq may cause serious side effects, including

  • Serious allergic (hypersensitivity) reactions. Serious allergic reactions have happened in some people who use Strensiq. Stop using Strensiq and go to the nearest hospital emergency room right away if you have any of the signs and symptoms of a serious allergic reaction, including
    • Difficulty breathing
    • Swelling of your eyes, lips, or tongue
    • Hives
    • Feeling faint
    • Nausea or vomiting
    • Dizziness
    • Itching of your lips, tongue, or throat
    • Choking sensation
  • Skin thickening or pits at the injection site (lipodystrophy). Lipodystrophy at the injection site has happened several months after using Strensiq
  • Calcium buildup in your eyes and kidneys. Your healthcare provider should check your eyes and kidneys while you use Strensiq

The most common side effects of Strensiq include local skin injection-site reactions (including red skin patches, bruising, color change, pain, itching, thinning, swelling, pits, and bumps) and calcium buildup in your eyes and kidneys.

Tell your doctor if you are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed.

These are not all of the possible side effects of Strensiq. For more information, ask your healthcare provider or pharmacist. Call your healthcare provider for medical advice about side effects. You may report side effects to the US Food and Drug Administration at 1-800-FDA-1088.

References:

  1. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(suppl 2):380-388.
  2. Fraser D. Hypophosphatasia. Am J Med. 1957;22(5):730-746.
  3. Whyte MP, Simmons JH, Moseley S, et al. Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019;7(2):93-105.
  4. Whyte MP, Greenberg CR, Salman NJ, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904-913.

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