CRYSVITA was effective in adults with TIO

Actor portrayal

Two clinical studies of adults with TIO have shown that
CRYSVITA can help by:

Increasing and maintaining phosphorus levels in the blood

Helping to heal osteomalacia

(weak or softened mature bone)

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Study design
Phosphorus levels
Osteomalacia

Study designs

The benefits and risks of treatment with CRYSVITA were studied in a total of 27 adults with TIO

Study 6

Number of patients: 14
Patient ages: 33 to 68 years
Length of treatment: 144 weeks

Study 7

Number of patients: 13
Patient ages: 41 to 73 years
Length of treatment: 88 weeks

INCREASING PHOSPHORUS

CRYSVITA increased phosphorus levels in the blood

In CRYSVITA clinical studies, adults with TIO had the level of phosphorus in their blood measured through week 144 (Study 6) and week 88 (Study 7).

Study 6

(Ages 33 to 68 years)

Line graph of increase in phosphorus serum levels

This chart shows the increase in phosphorus levels in the blood for people in Study 6 (14 adults, ages 33-68 years).

Phosphorus levels in the blood increased from 1.6 mg/dL at baseline to 2.64 mg/dL averaged across the midpoint of dose intervals through week 24 and were sustained through week 144.

For people receiving CRYSVITA in Study 7, serum phosphorus increased from 1.62 mg/dL at baseline to 2.63 mg/dL averaged across the midpoint of dose intervals through week 24. This increase was sustained through week 88 with CRYSVITA.

Study 6: 7 out of 14 people (50%) had serum phosphorus levels in the normal range averaged across the midpoint of dose intervals through week 24.

Study 7: 9 out of 13 people (69%) had serum phosphorus levels in the normal range averaged across the midpoint of dose intervals through week 24.

HEALING OSTEOMALACIA

CRYSVITA helped heal osteomalacia

Under normal circumstances, minerals like phosphorus are continuously added to bones. This process, called mineralization, helps keep bones hard. People with TIO don’t have enough phosphorus for bones to properly mineralize, which causes some parts of the bone to become “soft” and more likely to break.

In Study 6, a study of adults with TIO aged 33 to 68 years, small samples of bone were taken and examined for signs of healing of osteomalacia. Bone samples were taken before patients started treatment with CRYSVITA and then again after the patients were treated with CRYSVITA every 4 weeks for 48 weeks.

CRYSVITA helped improve bone mineralization in people with TIO.

Study 6
(14 adults, ages 33 to 68 years)

34%

Reduction in unmineralized or soft bone with CRYSVITA

Bone scans taken during Study 6 showed that some people receiving CRYSVITA saw healing in their bone abnormalities at week 48 through week 144.

In Studies 6 and 7, patients receiving CRYSVITA had a 34% reduction in unmineralized bone at week 48. Their bone biopsies showed a decrease in osteoid volume to bone volume (OV/BV), which is a measurement of unmineralized bone.

CRYSVITA reduced the bone damage caused by osteomalacia.